Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database.
Identifieur interne : 000F92 ( Main/Exploration ); précédent : 000F91; suivant : 000F93Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database.
Auteurs : Zaïda Koeks [Pays-Bas] ; Catherine L. Bladen [Royaume-Uni] ; David Salgado [France] ; Erik Van Zwet [Pays-Bas] ; Oksana Pogoryelova [Royaume-Uni] ; Grace Mcmacken [Royaume-Uni] ; Soledad Monges [Argentine] ; Maria Foncuberta [Argentine] ; Kyriaki Kekou [Grèce] ; Konstantina Kosma [Grèce] ; Hugh Dawkins [Australie] ; Leanne Lamont [Australie] ; Matthew I. Bellgard [Australie] ; Anna J. Roy [Belgique] ; Teodora Chamova [Bulgarie] ; Velina Guergueltcheva [Bulgarie] ; Sophelia Chan ; Lawrence Korngut [Canada] ; Craig Campbell [Canada] ; Yi Dai [République populaire de Chine] ; Jen Wang [République populaire de Chine] ; Nina Bariši [Croatie] ; Petr Brabec [République tchèque] ; Jaana L Hdetie [Finlande] ; Maggie C. Walter [Allemagne] ; Olivia Schreiber-Katz [Allemagne] ; Veronika Karcagi [Hongrie] ; Marta Garami [Hongrie] ; Agnes Herczegfalvi [Hongrie] ; Venkatarman Viswanathan [Inde] ; Farhad Bayat [Italie] ; Filippo Buccella [Italie] ; Alessandra Ferlini [Italie] ; En Kimura [Japon] ; Janneke C. Van Den Bergen [Pays-Bas] ; Miriam Rodrigues [Nouvelle-Zélande] ; Richard Roxburgh [Nouvelle-Zélande] ; Anna Lusakowska ; Anna Kostera-Pruszczyk ; Rosário Santos [Portugal] ; Elena Neagu [Roumanie] ; Svetlana Artemieva [Russie] ; Vedrana Milic Rasic [Serbie] ; Dina Vojinovic [Serbie] ; Manuel Posada [Espagne] ; Clemens Bloetzer [Suisse] ; Andrea Klein [Suisse] ; Jordi Díaz-Manera ; Eduard Gallardo [Suisse] ; A Ay E Karaduman [Turquie] ; Tunca Oznur [Turquie] ; Haluk Topalo Lu [Turquie] ; Rasha El Sherif [Égypte] ; Angela Stringer [Royaume-Uni] ; Andriy V. Shatillo [Ukraine] ; Ann S. Martin [États-Unis] ; Holly L. Peay [États-Unis] ; Jan Kirschner [Allemagne] ; Kevin M. Flanigan [États-Unis] ; Volker Straub [Royaume-Uni] ; Kate Bushby [Royaume-Uni] ; Christophe Béroud [France] ; Jan J. Verschuuren [Pays-Bas] ; Hanns Lochmüller [Royaume-Uni]Source :
- Journal of neuromuscular diseases [ 2214-3599 ] ; 2017.
Abstract
Recent short-term clinical trials in patients with Duchenne Muscular Dystrophy (DMD) have indicated greater disease variability in terms of progression than expected. In addition, as average life-expectancy increases, reliable data is required on clinical progression in the older DMD population.
DOI: 10.3233/JND-170280
PubMed: 29125504
Affiliations:
- Allemagne, Argentine, Australie, Belgique, Bulgarie, Canada, Croatie, Espagne, Finlande, France, Grèce, Hongrie, Inde, Italie, Japon, Nouvelle-Zélande, Pays-Bas, Portugal, Roumanie, Royaume-Uni, Russie, République populaire de Chine, République tchèque, Serbie, Suisse, Turquie, Ukraine, Égypte, États-Unis
- Alberta, Angleterre, Attique (région), Bavière, Canton de Berne, Canton de Vaud, Communauté de Madrid, District de Haute-Bavière, District fédéral central, Finlande occidentale, Grand Londres, Hongrie centrale, Latium, Moravie, New Jersey, Ohio, Provence-Alpes-Côte d'Azur, Région de Bruxelles-Capitale, Région de Kantō, Sofia-ville (oblast)
- Athènes, Berne, Brno, Bruxelles, Budapest, Calgary, Lausanne, Londres, Madrid, Marseille, Moscou, Munich, Pékin, Rome, Sofia, Tokyo, Turku
- Université Louis-et-Maximilien de Munich, Université de Berne, Université de Calgary
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Bladen</name><affiliation wicri:level="1"><nlm:affiliation>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne</wicri:regionArea><wicri:noRegion>Newcastle upon Tyne</wicri:noRegion></affiliation></author><author><name sortKey="Salgado, David" sort="Salgado, David" uniqKey="Salgado D" first="David" last="Salgado">David Salgado</name><affiliation wicri:level="3"><nlm:affiliation>AP-HM, Hôpital d'Enfants de la Timone, Département de Génétique Médicale et de Biologie Cellulaire, Marseille, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>AP-HM, Hôpital d'Enfants de la Timone, Département de Génétique Médicale et de Biologie Cellulaire, Marseille</wicri:regionArea><placeName><region type="region">Provence-Alpes-Côte d'Azur</region><region type="old region">Provence-Alpes-Côte d'Azur</region><settlement type="city">Marseille</settlement></placeName></affiliation></author><author><name sortKey="Van Zwet, Erik" sort="Van Zwet, Erik" uniqKey="Van Zwet E" first="Erik" last="Van Zwet">Erik Van Zwet</name><affiliation wicri:level="1"><nlm:affiliation>Leiden University Medical Center, Department of Medical Statistics, ZA Leiden, The Netherlands.</nlm:affiliation><country xml:lang="fr">Pays-Bas</country><wicri:regionArea>Leiden University Medical Center, Department of Medical Statistics, ZA Leiden</wicri:regionArea><wicri:noRegion>ZA Leiden</wicri:noRegion></affiliation></author><author><name sortKey="Pogoryelova, Oksana" sort="Pogoryelova, Oksana" uniqKey="Pogoryelova O" first="Oksana" last="Pogoryelova">Oksana Pogoryelova</name><affiliation wicri:level="1"><nlm:affiliation>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne</wicri:regionArea><wicri:noRegion>Newcastle upon Tyne</wicri:noRegion></affiliation></author><author><name sortKey="Mcmacken, Grace" sort="Mcmacken, Grace" uniqKey="Mcmacken G" first="Grace" last="Mcmacken">Grace Mcmacken</name><affiliation wicri:level="1"><nlm:affiliation>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne</wicri:regionArea><wicri:noRegion>Newcastle upon Tyne</wicri:noRegion></affiliation></author><author><name sortKey="Monges, Soledad" sort="Monges, Soledad" uniqKey="Monges S" first="Soledad" last="Monges">Soledad Monges</name><affiliation wicri:level="1"><nlm:affiliation>Hospital de Pediatría J. P. Garrahan, Pichincha, Argentina.</nlm:affiliation><country xml:lang="fr">Argentine</country><wicri:regionArea>Hospital de Pediatría J. P. Garrahan, Pichincha</wicri:regionArea><wicri:noRegion>Pichincha</wicri:noRegion></affiliation></author><author><name sortKey="Foncuberta, Maria" sort="Foncuberta, Maria" uniqKey="Foncuberta M" first="Maria" last="Foncuberta">Maria Foncuberta</name><affiliation wicri:level="1"><nlm:affiliation>Hospital de Pediatría J. P. Garrahan, Pichincha, Argentina.</nlm:affiliation><country xml:lang="fr">Argentine</country><wicri:regionArea>Hospital de Pediatría J. P. 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Bellgard</name><affiliation wicri:level="1"><nlm:affiliation>Centre for Comparative Genomics, Murdoch University, Murdoch, WA, Australia.</nlm:affiliation><country xml:lang="fr">Australie</country><wicri:regionArea>Centre for Comparative Genomics, Murdoch University, Murdoch, WA</wicri:regionArea><wicri:noRegion>WA</wicri:noRegion></affiliation></author><author><name sortKey="Roy, Anna J" sort="Roy, Anna J" uniqKey="Roy A" first="Anna J" last="Roy">Anna J. Roy</name><affiliation wicri:level="3"><nlm:affiliation>WIV-ISP, Brussels, Belgium.</nlm:affiliation><country xml:lang="fr">Belgique</country><wicri:regionArea>WIV-ISP, Brussels</wicri:regionArea><placeName><settlement type="city">Bruxelles</settlement><region nuts="2">Région de Bruxelles-Capitale</region></placeName></affiliation></author><author><name sortKey="Chamova, Teodora" sort="Chamova, Teodora" uniqKey="Chamova T" first="Teodora" last="Chamova">Teodora Chamova</name><affiliation wicri:level="3"><nlm:affiliation>Department of Neurology, Medical University-Sofia, Sofia, Bulgaria.</nlm:affiliation><country xml:lang="fr">Bulgarie</country><wicri:regionArea>Department of Neurology, Medical University-Sofia, Sofia</wicri:regionArea><placeName><settlement type="city">Sofia</settlement><region nuts="2">Sofia-ville (oblast)</region></placeName></affiliation></author><author><name sortKey="Guergueltcheva, Velina" sort="Guergueltcheva, Velina" uniqKey="Guergueltcheva V" first="Velina" last="Guergueltcheva">Velina Guergueltcheva</name><affiliation wicri:level="3"><nlm:affiliation>Clinic of Neurology, University Hospital Sofiamed, Sofia, Bulgaria.</nlm:affiliation><country xml:lang="fr">Bulgarie</country><wicri:regionArea>Clinic of Neurology, University Hospital Sofiamed, Sofia</wicri:regionArea><placeName><settlement type="city">Sofia</settlement><region nuts="2">Sofia-ville (oblast)</region></placeName></affiliation></author><author><name sortKey="Chan, Sophelia" sort="Chan, Sophelia" uniqKey="Chan S" first="Sophelia" last="Chan">Sophelia Chan</name><affiliation><nlm:affiliation>Department of Paediatrics and Adolescent Medicine, Queen Mary Hospital, University of Hong Kong.</nlm:affiliation><wicri:noCountry code="subField">University of Hong Kong</wicri:noCountry></affiliation></author><author><name sortKey="Korngut, Lawrence" sort="Korngut, Lawrence" uniqKey="Korngut L" first="Lawrence" last="Korngut">Lawrence Korngut</name><affiliation wicri:level="4"><nlm:affiliation>Department of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, South Health Campus, Calgary, AB, Canada.</nlm:affiliation><country xml:lang="fr">Canada</country><wicri:regionArea>Department of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, South Health Campus, Calgary, AB</wicri:regionArea><orgName type="university">Université de Calgary</orgName><placeName><settlement type="city">Calgary</settlement><region type="state">Alberta</region></placeName></affiliation></author><author><name sortKey="Campbell, Craig" sort="Campbell, Craig" uniqKey="Campbell C" first="Craig" last="Campbell">Craig Campbell</name><affiliation wicri:level="1"><nlm:affiliation>Department of Paediatrics, Clinical Neurological Sciences & Epidemiology, Western University, London, ON, Canada.</nlm:affiliation><country xml:lang="fr">Canada</country><wicri:regionArea>Department of Paediatrics, Clinical Neurological Sciences & Epidemiology, Western University, London, ON</wicri:regionArea><wicri:noRegion>ON</wicri:noRegion></affiliation></author><author><name sortKey="Dai, Yi" sort="Dai, Yi" uniqKey="Dai Y" first="Yi" last="Dai">Yi Dai</name><affiliation wicri:level="3"><nlm:affiliation>Department of Neurology, Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, Beijing, China.</nlm:affiliation><country xml:lang="fr">République populaire de Chine</country><wicri:regionArea>Department of Neurology, Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, Beijing</wicri:regionArea><placeName><settlement type="city">Pékin</settlement></placeName></affiliation></author><author><name sortKey="Wang, Jen" sort="Wang, Jen" uniqKey="Wang J" first="Jen" last="Wang">Jen Wang</name><affiliation wicri:level="1"><nlm:affiliation>China DMD Care and Support Association c/o China Dolls, Xicheng district, China.</nlm:affiliation><country xml:lang="fr">République populaire de Chine</country><wicri:regionArea>China DMD Care and Support Association c/o China Dolls, Xicheng district</wicri:regionArea><wicri:noRegion>Xicheng district</wicri:noRegion></affiliation></author><author><name sortKey="Barisi, Nina" sort="Barisi, Nina" uniqKey="Barisi N" first="Nina" last="Bariši">Nina Bariši</name><affiliation wicri:level="1"><nlm:affiliation>Division of Paediatric Neurology, University Hospital Centre Zagreb (KBC Zagreb) University of Zagreb Medical School, Croatia.</nlm:affiliation><country xml:lang="fr">Croatie</country><wicri:regionArea>Division of Paediatric Neurology, University Hospital Centre Zagreb (KBC Zagreb) University of Zagreb Medical School</wicri:regionArea><wicri:noRegion>University Hospital Centre Zagreb (KBC Zagreb) University of Zagreb Medical School</wicri:noRegion></affiliation></author><author><name sortKey="Brabec, Petr" sort="Brabec, Petr" uniqKey="Brabec P" first="Petr" last="Brabec">Petr Brabec</name><affiliation wicri:level="3"><nlm:affiliation>Institute for Biostatistic and Analyses, Masaryk University, Brno, Czech Republic.</nlm:affiliation><country xml:lang="fr">République tchèque</country><wicri:regionArea>Institute for Biostatistic and Analyses, Masaryk University, Brno</wicri:regionArea><placeName><settlement type="city">Brno</settlement><region>Moravie</region></placeName></affiliation></author><author><name sortKey="L Hdetie, Jaana" sort="L Hdetie, Jaana" uniqKey="L Hdetie J" first="Jaana" last="L Hdetie">Jaana L Hdetie</name><affiliation wicri:level="3"><nlm:affiliation>Department of Child Neurology, Turku University Central Hospital, Turku, Finland.</nlm:affiliation><country xml:lang="fr">Finlande</country><wicri:regionArea>Department of Child Neurology, Turku University Central Hospital, Turku</wicri:regionArea><placeName><settlement type="city">Turku</settlement><region type="région" nuts="2">Finlande occidentale</region></placeName></affiliation></author><author><name sortKey="Walter, Maggie C" sort="Walter, Maggie C" uniqKey="Walter M" first="Maggie C" last="Walter">Maggie C. Walter</name><affiliation wicri:level="4"><nlm:affiliation>Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany.</nlm:affiliation><country xml:lang="fr">Allemagne</country><wicri:regionArea>Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich</wicri:regionArea><placeName><region type="land" nuts="1">Bavière</region><region type="district" nuts="2">District de Haute-Bavière</region><settlement type="city">Munich</settlement><settlement type="city">Munich</settlement></placeName><orgName type="university">Université Louis-et-Maximilien de Munich</orgName></affiliation></author><author><name sortKey="Schreiber Katz, Olivia" sort="Schreiber Katz, Olivia" uniqKey="Schreiber Katz O" first="Olivia" last="Schreiber-Katz">Olivia Schreiber-Katz</name><affiliation wicri:level="4"><nlm:affiliation>Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany.</nlm:affiliation><country xml:lang="fr">Allemagne</country><wicri:regionArea>Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich</wicri:regionArea><placeName><region type="land" nuts="1">Bavière</region><region type="district" nuts="2">District de Haute-Bavière</region><settlement type="city">Munich</settlement><settlement type="city">Munich</settlement></placeName><orgName type="university">Université Louis-et-Maximilien de Munich</orgName></affiliation></author><author><name sortKey="Karcagi, Veronika" sort="Karcagi, Veronika" uniqKey="Karcagi V" first="Veronika" last="Karcagi">Veronika Karcagi</name><affiliation wicri:level="3"><nlm:affiliation>NIEH, Department of Molecular Genetics and Diagnostics Albert Florian str 2-6, Budapest, Hungary.</nlm:affiliation><country xml:lang="fr">Hongrie</country><wicri:regionArea>NIEH, Department of Molecular Genetics and Diagnostics Albert Florian str 2-6, Budapest</wicri:regionArea><placeName><settlement type="city">Budapest</settlement><region nuts="2">Hongrie centrale</region></placeName></affiliation></author><author><name sortKey="Garami, Marta" sort="Garami, Marta" uniqKey="Garami M" first="Marta" last="Garami">Marta Garami</name><affiliation wicri:level="3"><nlm:affiliation>NIEH, Department of Molecular Genetics and Diagnostics Albert Florian str 2-6, Budapest, Hungary.</nlm:affiliation><country xml:lang="fr">Hongrie</country><wicri:regionArea>NIEH, Department of Molecular Genetics and Diagnostics Albert Florian str 2-6, Budapest</wicri:regionArea><placeName><settlement type="city">Budapest</settlement><region nuts="2">Hongrie centrale</region></placeName></affiliation></author><author><name sortKey="Herczegfalvi, Agnes" sort="Herczegfalvi, Agnes" uniqKey="Herczegfalvi A" first="Agnes" last="Herczegfalvi">Agnes Herczegfalvi</name><affiliation wicri:level="1"><nlm:affiliation>Semmelweis Medical University, II. Department of Paediatric Neurology, Budapest, Tűzoltó u, Hungary.</nlm:affiliation><country xml:lang="fr">Hongrie</country><wicri:regionArea>Semmelweis Medical University, II. Department of Paediatric Neurology, Budapest, Tűzoltó u</wicri:regionArea><wicri:noRegion>Tűzoltó u</wicri:noRegion></affiliation></author><author><name sortKey="Viswanathan, Venkatarman" sort="Viswanathan, Venkatarman" uniqKey="Viswanathan V" first="Venkatarman" last="Viswanathan">Venkatarman Viswanathan</name><affiliation wicri:level="1"><nlm:affiliation>Kanchi Kamakoti CHILDS Trust and Apollo Children's Hospitals, Chennai, India.</nlm:affiliation><country xml:lang="fr">Inde</country><wicri:regionArea>Kanchi Kamakoti CHILDS Trust and Apollo Children's Hospitals, Chennai</wicri:regionArea><wicri:noRegion>Chennai</wicri:noRegion></affiliation></author><author><name sortKey="Bayat, Farhad" sort="Bayat, Farhad" uniqKey="Bayat F" first="Farhad" last="Bayat">Farhad Bayat</name><affiliation wicri:level="3"><nlm:affiliation>Pasteur Institute of Iran, Karaj complex, Tehran, Iran. Parent Project Onlus, Roma, Italy.</nlm:affiliation><country xml:lang="fr">Italie</country><wicri:regionArea>Pasteur Institute of Iran, Karaj complex, Tehran, Iran. Parent Project Onlus, Roma</wicri:regionArea><placeName><settlement type="city">Rome</settlement><region nuts="2">Latium</region></placeName></affiliation></author><author><name sortKey="Buccella, Filippo" sort="Buccella, Filippo" uniqKey="Buccella F" first="Filippo" last="Buccella">Filippo Buccella</name><affiliation wicri:level="3"><nlm:affiliation>Parent Project Onlus, Roma, Italy.</nlm:affiliation><country xml:lang="fr">Italie</country><wicri:regionArea>Parent Project Onlus, Roma</wicri:regionArea><placeName><settlement type="city">Rome</settlement><region nuts="2">Latium</region></placeName></affiliation></author><author><name sortKey="Ferlini, Alessandra" sort="Ferlini, Alessandra" uniqKey="Ferlini A" first="Alessandra" last="Ferlini">Alessandra Ferlini</name><affiliation wicri:level="1"><nlm:affiliation>Department of Reproduction and Growth, Department of Medical Sciences, OSPFE, University of Ferrara, Italy.</nlm:affiliation><country xml:lang="fr">Italie</country><wicri:regionArea>Department of Reproduction and Growth, Department of Medical Sciences, OSPFE, University of Ferrara</wicri:regionArea><wicri:noRegion>University of Ferrara</wicri:noRegion></affiliation></author><author><name sortKey="Kimura, En" sort="Kimura, En" uniqKey="Kimura E" first="En" last="Kimura">En Kimura</name><affiliation wicri:level="3"><nlm:affiliation>214-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.</nlm:affiliation><country xml:lang="fr">Japon</country><wicri:regionArea>214-1-1 Ogawa-Higashi, Kodaira, Tokyo</wicri:regionArea><placeName><settlement type="city">Tokyo</settlement><region type="région">Région de Kantō</region></placeName></affiliation></author><author><name sortKey="Van Den Bergen, Janneke C" sort="Van Den Bergen, Janneke C" uniqKey="Van Den Bergen J" first="Janneke C" last="Van Den Bergen">Janneke C. Van Den Bergen</name><affiliation wicri:level="1"><nlm:affiliation>Leiden University Medical Center, Department of Neurology, ZA Leiden, The Netherlands.</nlm:affiliation><country xml:lang="fr">Pays-Bas</country><wicri:regionArea>Leiden University Medical Center, Department of Neurology, ZA Leiden</wicri:regionArea><wicri:noRegion>ZA Leiden</wicri:noRegion></affiliation></author><author><name sortKey="Rodrigues, Miriam" sort="Rodrigues, Miriam" uniqKey="Rodrigues M" first="Miriam" last="Rodrigues">Miriam Rodrigues</name><affiliation wicri:level="1"><nlm:affiliation>Department of Neurology, Auckland DHB, Auckland, New Zealand.</nlm:affiliation><country xml:lang="fr">Nouvelle-Zélande</country><wicri:regionArea>Department of Neurology, Auckland DHB, Auckland</wicri:regionArea><wicri:noRegion>Auckland</wicri:noRegion></affiliation></author><author><name sortKey="Roxburgh, Richard" sort="Roxburgh, Richard" uniqKey="Roxburgh R" first="Richard" last="Roxburgh">Richard Roxburgh</name><affiliation wicri:level="1"><nlm:affiliation>Department of Neurology, Auckland DHB, Auckland, New Zealand.</nlm:affiliation><country xml:lang="fr">Nouvelle-Zélande</country><wicri:regionArea>Department of Neurology, Auckland DHB, Auckland</wicri:regionArea><wicri:noRegion>Auckland</wicri:noRegion></affiliation></author><author><name sortKey="Lusakowska, Anna" sort="Lusakowska, Anna" uniqKey="Lusakowska A" first="Anna" last="Lusakowska">Anna Lusakowska</name><affiliation><nlm:affiliation>Department of Neurology, Medical University of Warsaw, Warsaw. Poland.</nlm:affiliation><wicri:noCountry code="subField">Warsaw</wicri:noCountry></affiliation></author><author><name sortKey="Kostera Pruszczyk, Anna" sort="Kostera Pruszczyk, Anna" uniqKey="Kostera Pruszczyk A" first="Anna" last="Kostera-Pruszczyk">Anna Kostera-Pruszczyk</name><affiliation><nlm:affiliation>Department of Neurology, Medical University of Warsaw, Warsaw. Poland.</nlm:affiliation><wicri:noCountry code="subField">Warsaw</wicri:noCountry></affiliation></author><author><name sortKey="Santos, Rosario" sort="Santos, Rosario" uniqKey="Santos R" first="Rosário" last="Santos">Rosário Santos</name><affiliation wicri:level="1"><nlm:affiliation>Centro de Genética Médica Jacinto Magalhães, Porto, Portugal.</nlm:affiliation><country xml:lang="fr">Portugal</country><wicri:regionArea>Centro de Genética Médica Jacinto Magalhães, Porto</wicri:regionArea><wicri:noRegion>Porto</wicri:noRegion></affiliation></author><author><name sortKey="Neagu, Elena" sort="Neagu, Elena" uniqKey="Neagu E" first="Elena" last="Neagu">Elena Neagu</name><affiliation wicri:level="1"><nlm:affiliation>National Institute of Legal Medicine "Mina Minovici" - Genetics Laboratory, Bucharest, Romania.</nlm:affiliation><country xml:lang="fr">Roumanie</country><wicri:regionArea>National Institute of Legal Medicine "Mina Minovici" - Genetics Laboratory, Bucharest</wicri:regionArea><wicri:noRegion>Bucharest</wicri:noRegion></affiliation></author><author><name sortKey="Artemieva, Svetlana" sort="Artemieva, Svetlana" uniqKey="Artemieva S" first="Svetlana" last="Artemieva">Svetlana Artemieva</name><affiliation wicri:level="3"><nlm:affiliation>Moscow Institute of Pediatrics, Moscow, Russia.</nlm:affiliation><country xml:lang="fr">Russie</country><wicri:regionArea>Moscow Institute of Pediatrics, Moscow</wicri:regionArea><placeName><settlement type="city">Moscou</settlement><region>District fédéral central</region></placeName></affiliation></author><author><name sortKey="Rasic, Vedrana Milic" sort="Rasic, Vedrana Milic" uniqKey="Rasic V" first="Vedrana Milic" last="Rasic">Vedrana Milic Rasic</name><affiliation wicri:level="1"><nlm:affiliation>Clinic for Neurology and Psychiatry for Children and Youth, Faculty of Medicine, University of Belgrade, Belgrade, Serbia.</nlm:affiliation><country xml:lang="fr">Serbie</country><wicri:regionArea>Clinic for Neurology and Psychiatry for Children and Youth, Faculty of Medicine, University of Belgrade, Belgrade</wicri:regionArea><wicri:noRegion>Belgrade</wicri:noRegion></affiliation></author><author><name sortKey="Vojinovic, Dina" sort="Vojinovic, Dina" uniqKey="Vojinovic D" first="Dina" last="Vojinovic">Dina Vojinovic</name><affiliation wicri:level="1"><nlm:affiliation>Clinic for Neurology and Psychiatry for Children and Youth, Faculty of Medicine, University of Belgrade, Belgrade, Serbia.</nlm:affiliation><country xml:lang="fr">Serbie</country><wicri:regionArea>Clinic for Neurology and Psychiatry for Children and Youth, Faculty of Medicine, University of Belgrade, Belgrade</wicri:regionArea><wicri:noRegion>Belgrade</wicri:noRegion></affiliation></author><author><name sortKey="Posada, Manuel" sort="Posada, Manuel" uniqKey="Posada M" first="Manuel" last="Posada">Manuel Posada</name><affiliation wicri:level="3"><nlm:affiliation>Institute of Rare Diseases Research, SpainRDR and CIBERER, Institute of Health Carlos III, Madrid, Spain.</nlm:affiliation><country xml:lang="fr">Espagne</country><wicri:regionArea>Institute of Rare Diseases Research, SpainRDR and CIBERER, Institute of Health Carlos III, Madrid</wicri:regionArea><placeName><settlement type="city">Madrid</settlement><region nuts="2" type="region">Communauté de Madrid</region></placeName></affiliation></author><author><name sortKey="Bloetzer, Clemens" sort="Bloetzer, Clemens" uniqKey="Bloetzer C" first="Clemens" last="Bloetzer">Clemens Bloetzer</name><affiliation wicri:level="4"><nlm:affiliation>Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.</nlm:affiliation><country xml:lang="fr">Suisse</country><wicri:regionArea>Institute of Social and Preventive Medicine, University of Bern, Bern</wicri:regionArea><placeName><settlement type="city">Berne</settlement><region type="région" nuts="3">Canton de Berne</region><settlement type="city">Berne</settlement></placeName><orgName type="university">Université de Berne</orgName></affiliation></author><author><name sortKey="Klein, Andrea" sort="Klein, Andrea" uniqKey="Klein A" first="Andrea" last="Klein">Andrea Klein</name><affiliation wicri:level="3"><nlm:affiliation>Paediatric Neurology and Neurorehabilitation Unit, Lausanne University Hospital, Lausanne, Switzerland.</nlm:affiliation><country xml:lang="fr">Suisse</country><wicri:regionArea>Paediatric Neurology and Neurorehabilitation Unit, Lausanne University Hospital, Lausanne</wicri:regionArea><placeName><settlement type="city">Lausanne</settlement><region nuts="3" type="region">Canton de Vaud</region></placeName></affiliation></author><author><name sortKey="Diaz Manera, Jordi" sort="Diaz Manera, Jordi" uniqKey="Diaz Manera J" first="Jordi" last="Díaz-Manera">Jordi Díaz-Manera</name><affiliation><nlm:affiliation>Unitat de Malalties Neuromusculars, Servei de Neurologia, Hospital de la Santa Creu i Sant Pau de Barcelona.</nlm:affiliation><wicri:noCountry code="subField">Hospital de la Santa Creu i Sant Pau de Barcelona</wicri:noCountry></affiliation></author><author><name sortKey="Gallardo, Eduard" sort="Gallardo, Eduard" uniqKey="Gallardo E" first="Eduard" last="Gallardo">Eduard Gallardo</name><affiliation wicri:level="3"><nlm:affiliation>Paediatric Neurology and Neurorehabilitation Unit, Lausanne University Hospital, Lausanne, Switzerland.</nlm:affiliation><country xml:lang="fr">Suisse</country><wicri:regionArea>Paediatric Neurology and Neurorehabilitation Unit, Lausanne University Hospital, Lausanne</wicri:regionArea><placeName><settlement type="city">Lausanne</settlement><region nuts="3" type="region">Canton de Vaud</region></placeName></affiliation></author><author><name sortKey="Karaduman, A Ay E" sort="Karaduman, A Ay E" uniqKey="Karaduman A" first="A Ay E" last="Karaduman">A Ay E Karaduman</name><affiliation wicri:level="1"><nlm:affiliation>Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara, Turkey.</nlm:affiliation><country xml:lang="fr">Turquie</country><wicri:regionArea>Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara</wicri:regionArea><wicri:noRegion>Ankara</wicri:noRegion></affiliation></author><author><name sortKey="Oznur, Tunca" sort="Oznur, Tunca" uniqKey="Oznur T" first="Tunca" last="Oznur">Tunca Oznur</name><affiliation wicri:level="1"><nlm:affiliation>Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara, Turkey.</nlm:affiliation><country xml:lang="fr">Turquie</country><wicri:regionArea>Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara</wicri:regionArea><wicri:noRegion>Ankara</wicri:noRegion></affiliation></author><author><name sortKey="Topalo Lu, Haluk" sort="Topalo Lu, Haluk" uniqKey="Topalo Lu H" first="Haluk" last="Topalo Lu">Haluk Topalo Lu</name><affiliation wicri:level="1"><nlm:affiliation>Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara, Turkey.</nlm:affiliation><country xml:lang="fr">Turquie</country><wicri:regionArea>Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara</wicri:regionArea><wicri:noRegion>Ankara</wicri:noRegion></affiliation></author><author><name sortKey="El Sherif, Rasha" sort="El Sherif, Rasha" uniqKey="El Sherif R" first="Rasha" last="El Sherif">Rasha El Sherif</name><affiliation wicri:level="1"><nlm:affiliation>Neurology & Neurogenic Unit, Egypt Air Hospital, Ain Shams University, Egypt.</nlm:affiliation><country xml:lang="fr">Égypte</country><wicri:regionArea>Neurology & Neurogenic Unit, Egypt Air Hospital, Ain Shams University</wicri:regionArea><wicri:noRegion>Ain Shams University</wicri:noRegion></affiliation></author><author><name sortKey="Stringer, Angela" sort="Stringer, Angela" uniqKey="Stringer A" first="Angela" last="Stringer">Angela Stringer</name><affiliation wicri:level="3"><nlm:affiliation>Action Duchenne, Epicentre, London, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Action Duchenne, Epicentre, London</wicri:regionArea><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region></placeName></affiliation></author><author><name sortKey="Shatillo, Andriy V" sort="Shatillo, Andriy V" uniqKey="Shatillo A" first="Andriy V" last="Shatillo">Andriy V. Shatillo</name><affiliation wicri:level="1"><nlm:affiliation>Institute of Neurology, Psychiatry and Narcology of NAMS, Kharkiv, Ukraine.</nlm:affiliation><country xml:lang="fr">Ukraine</country><wicri:regionArea>Institute of Neurology, Psychiatry and Narcology of NAMS, Kharkiv</wicri:regionArea><wicri:noRegion>Kharkiv</wicri:noRegion></affiliation></author><author><name sortKey="Martin, Ann S" sort="Martin, Ann S" uniqKey="Martin A" first="Ann S" last="Martin">Ann S. Martin</name><affiliation wicri:level="2"><nlm:affiliation>DuchenneConnect, Hackensack, NJ, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>DuchenneConnect, Hackensack, NJ</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="Peay, Holly L" sort="Peay, Holly L" uniqKey="Peay H" first="Holly L" last="Peay">Holly L. Peay</name><affiliation wicri:level="2"><nlm:affiliation>DuchenneConnect, Hackensack, NJ, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>DuchenneConnect, Hackensack, NJ</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="Kirschner, Jan" sort="Kirschner, Jan" uniqKey="Kirschner J" first="Jan" last="Kirschner">Jan Kirschner</name><affiliation wicri:level="1"><nlm:affiliation>University Medical Center Freiburg, Germany.</nlm:affiliation><country xml:lang="fr">Allemagne</country><wicri:regionArea>University Medical Center Freiburg</wicri:regionArea></affiliation></author><author><name sortKey="Flanigan, Kevin M" sort="Flanigan, Kevin M" uniqKey="Flanigan K" first="Kevin M" last="Flanigan">Kevin M. Flanigan</name><affiliation wicri:level="2"><nlm:affiliation>Center for Gene Therapy, The Research Institute, Nationwide Children's Hospital, Columbus, OH, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>Center for Gene Therapy, The Research Institute, Nationwide Children's Hospital, Columbus, OH</wicri:regionArea><placeName><region type="state">Ohio</region></placeName></affiliation></author><author><name sortKey="Straub, Volker" sort="Straub, Volker" uniqKey="Straub V" first="Volker" last="Straub">Volker Straub</name><affiliation wicri:level="1"><nlm:affiliation>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne</wicri:regionArea><wicri:noRegion>Newcastle upon Tyne</wicri:noRegion></affiliation></author><author><name sortKey="Bushby, Kate" sort="Bushby, Kate" uniqKey="Bushby K" first="Kate" last="Bushby">Kate Bushby</name><affiliation wicri:level="1"><nlm:affiliation>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne</wicri:regionArea><wicri:noRegion>Newcastle upon Tyne</wicri:noRegion></affiliation></author><author><name sortKey="Beroud, Christophe" sort="Beroud, Christophe" uniqKey="Beroud C" first="Christophe" last="Béroud">Christophe Béroud</name><affiliation wicri:level="3"><nlm:affiliation>AP-HM, Hôpital d'Enfants de la Timone, Département de Génétique Médicale et de Biologie Cellulaire, Marseille, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>AP-HM, Hôpital d'Enfants de la Timone, Département de Génétique Médicale et de Biologie Cellulaire, Marseille</wicri:regionArea><placeName><region type="region">Provence-Alpes-Côte d'Azur</region><region type="old region">Provence-Alpes-Côte d'Azur</region><settlement type="city">Marseille</settlement></placeName></affiliation></author><author><name sortKey="Verschuuren, Jan J" sort="Verschuuren, Jan J" uniqKey="Verschuuren J" first="Jan J" last="Verschuuren">Jan J. Verschuuren</name><affiliation wicri:level="1"><nlm:affiliation>Leiden University Medical Center, Department of Neurology, ZA Leiden, The Netherlands.</nlm:affiliation><country xml:lang="fr">Pays-Bas</country><wicri:regionArea>Leiden University Medical Center, Department of Neurology, ZA Leiden</wicri:regionArea><wicri:noRegion>ZA Leiden</wicri:noRegion></affiliation></author><author><name sortKey="Lochmuller, Hanns" sort="Lochmuller, Hanns" uniqKey="Lochmuller H" first="Hanns" last="Lochmüller">Hanns Lochmüller</name><affiliation wicri:level="1"><nlm:affiliation>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne</wicri:regionArea><wicri:noRegion>Newcastle upon Tyne</wicri:noRegion></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PubMed</idno><date when="2017">2017</date><idno type="RBID">pubmed:29125504</idno><idno type="pmid">29125504</idno><idno type="doi">10.3233/JND-170280</idno><idno type="wicri:Area/PubMed/Corpus">000137</idno><idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Corpus" wicri:corpus="PubMed">000137</idno><idno type="wicri:Area/PubMed/Curation">000137</idno><idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Curation">000137</idno><idno type="wicri:Area/PubMed/Checkpoint">000137</idno><idno type="wicri:explorRef" wicri:stream="Checkpoint" wicri:step="PubMed">000137</idno><idno type="wicri:Area/Ncbi/Merge">005196</idno><idno type="wicri:Area/Ncbi/Curation">005196</idno><idno type="wicri:Area/Ncbi/Checkpoint">005196</idno><idno type="wicri:doubleKey">2214-3599:2017:Koeks Z:clinical:outcomes:in</idno><idno type="wicri:Area/Main/Merge">000F87</idno><idno type="wicri:Area/Main/Curation">000F92</idno><idno type="wicri:Area/Main/Exploration">000F92</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en">Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database.</title><author><name sortKey="Koeks, Zaida" sort="Koeks, Zaida" uniqKey="Koeks Z" first="Zaïda" last="Koeks">Zaïda Koeks</name><affiliation wicri:level="1"><nlm:affiliation>Leiden University Medical Center, Department of Neurology, ZA Leiden, The Netherlands.</nlm:affiliation><country xml:lang="fr">Pays-Bas</country><wicri:regionArea>Leiden University Medical Center, Department of Neurology, ZA Leiden</wicri:regionArea><wicri:noRegion>ZA Leiden</wicri:noRegion></affiliation></author><author><name sortKey="Bladen, Catherine L" sort="Bladen, Catherine L" uniqKey="Bladen C" first="Catherine L" last="Bladen">Catherine L. Bladen</name><affiliation wicri:level="1"><nlm:affiliation>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne</wicri:regionArea><wicri:noRegion>Newcastle upon Tyne</wicri:noRegion></affiliation></author><author><name sortKey="Salgado, David" sort="Salgado, David" uniqKey="Salgado D" first="David" last="Salgado">David Salgado</name><affiliation wicri:level="3"><nlm:affiliation>AP-HM, Hôpital d'Enfants de la Timone, Département de Génétique Médicale et de Biologie Cellulaire, Marseille, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>AP-HM, Hôpital d'Enfants de la Timone, Département de Génétique Médicale et de Biologie Cellulaire, Marseille</wicri:regionArea><placeName><region type="region">Provence-Alpes-Côte d'Azur</region><region type="old region">Provence-Alpes-Côte d'Azur</region><settlement type="city">Marseille</settlement></placeName></affiliation></author><author><name sortKey="Van Zwet, Erik" sort="Van Zwet, Erik" uniqKey="Van Zwet E" first="Erik" last="Van Zwet">Erik Van Zwet</name><affiliation wicri:level="1"><nlm:affiliation>Leiden University Medical Center, Department of Medical Statistics, ZA Leiden, The Netherlands.</nlm:affiliation><country xml:lang="fr">Pays-Bas</country><wicri:regionArea>Leiden University Medical Center, Department of Medical Statistics, ZA Leiden</wicri:regionArea><wicri:noRegion>ZA Leiden</wicri:noRegion></affiliation></author><author><name sortKey="Pogoryelova, Oksana" sort="Pogoryelova, Oksana" uniqKey="Pogoryelova O" first="Oksana" last="Pogoryelova">Oksana Pogoryelova</name><affiliation wicri:level="1"><nlm:affiliation>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne</wicri:regionArea><wicri:noRegion>Newcastle upon Tyne</wicri:noRegion></affiliation></author><author><name sortKey="Mcmacken, Grace" sort="Mcmacken, Grace" uniqKey="Mcmacken G" first="Grace" last="Mcmacken">Grace Mcmacken</name><affiliation wicri:level="1"><nlm:affiliation>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne</wicri:regionArea><wicri:noRegion>Newcastle upon Tyne</wicri:noRegion></affiliation></author><author><name sortKey="Monges, Soledad" sort="Monges, Soledad" uniqKey="Monges S" first="Soledad" last="Monges">Soledad Monges</name><affiliation wicri:level="1"><nlm:affiliation>Hospital de Pediatría J. P. Garrahan, Pichincha, Argentina.</nlm:affiliation><country xml:lang="fr">Argentine</country><wicri:regionArea>Hospital de Pediatría J. P. Garrahan, Pichincha</wicri:regionArea><wicri:noRegion>Pichincha</wicri:noRegion></affiliation></author><author><name sortKey="Foncuberta, Maria" sort="Foncuberta, Maria" uniqKey="Foncuberta M" first="Maria" last="Foncuberta">Maria Foncuberta</name><affiliation wicri:level="1"><nlm:affiliation>Hospital de Pediatría J. P. Garrahan, Pichincha, Argentina.</nlm:affiliation><country xml:lang="fr">Argentine</country><wicri:regionArea>Hospital de Pediatría J. P. Garrahan, Pichincha</wicri:regionArea><wicri:noRegion>Pichincha</wicri:noRegion></affiliation></author><author><name sortKey="Kekou, Kyriaki" sort="Kekou, Kyriaki" uniqKey="Kekou K" first="Kyriaki" last="Kekou">Kyriaki Kekou</name><affiliation wicri:level="3"><nlm:affiliation>Department of Medical Genetics, Medical School, University of Athens, Choremio Research Laboratory, St. Sophia's Children's Hospital Thinon and Levadia Goudi, Athens, Greece.</nlm:affiliation><country xml:lang="fr">Grèce</country><wicri:regionArea>Department of Medical Genetics, Medical School, University of Athens, Choremio Research Laboratory, St. Sophia's Children's Hospital Thinon and Levadia Goudi, Athens</wicri:regionArea><placeName><settlement type="city">Athènes</settlement><region nuts="2" type="region">Attique (région)</region></placeName></affiliation></author><author><name sortKey="Kosma, Konstantina" sort="Kosma, Konstantina" uniqKey="Kosma K" first="Konstantina" last="Kosma">Konstantina Kosma</name><affiliation wicri:level="3"><nlm:affiliation>Department of Medical Genetics, Medical School, University of Athens, Choremio Research Laboratory, St. Sophia's Children's Hospital Thinon and Levadia Goudi, Athens, Greece.</nlm:affiliation><country xml:lang="fr">Grèce</country><wicri:regionArea>Department of Medical Genetics, Medical School, University of Athens, Choremio Research Laboratory, St. Sophia's Children's Hospital Thinon and Levadia Goudi, Athens</wicri:regionArea><placeName><settlement type="city">Athènes</settlement><region nuts="2" type="region">Attique (région)</region></placeName></affiliation></author><author><name sortKey="Dawkins, Hugh" sort="Dawkins, Hugh" uniqKey="Dawkins H" first="Hugh" last="Dawkins">Hugh Dawkins</name><affiliation wicri:level="1"><nlm:affiliation>Office of Population Health Genomics, Department of Health, Perth, WA, Australia.</nlm:affiliation><country xml:lang="fr">Australie</country><wicri:regionArea>Office of Population Health Genomics, Department of Health, Perth, WA</wicri:regionArea><wicri:noRegion>WA</wicri:noRegion></affiliation></author><author><name sortKey="Lamont, Leanne" sort="Lamont, Leanne" uniqKey="Lamont L" first="Leanne" last="Lamont">Leanne Lamont</name><affiliation wicri:level="1"><nlm:affiliation>Office of Population Health Genomics, Department of Health, Perth, WA, Australia.</nlm:affiliation><country xml:lang="fr">Australie</country><wicri:regionArea>Office of Population Health Genomics, Department of Health, Perth, WA</wicri:regionArea><wicri:noRegion>WA</wicri:noRegion></affiliation></author><author><name sortKey="Bellgard, Matthew I" sort="Bellgard, Matthew I" uniqKey="Bellgard M" first="Matthew I" last="Bellgard">Matthew I. Bellgard</name><affiliation wicri:level="1"><nlm:affiliation>Centre for Comparative Genomics, Murdoch University, Murdoch, WA, Australia.</nlm:affiliation><country xml:lang="fr">Australie</country><wicri:regionArea>Centre for Comparative Genomics, Murdoch University, Murdoch, WA</wicri:regionArea><wicri:noRegion>WA</wicri:noRegion></affiliation></author><author><name sortKey="Roy, Anna J" sort="Roy, Anna J" uniqKey="Roy A" first="Anna J" last="Roy">Anna J. Roy</name><affiliation wicri:level="3"><nlm:affiliation>WIV-ISP, Brussels, Belgium.</nlm:affiliation><country xml:lang="fr">Belgique</country><wicri:regionArea>WIV-ISP, Brussels</wicri:regionArea><placeName><settlement type="city">Bruxelles</settlement><region nuts="2">Région de Bruxelles-Capitale</region></placeName></affiliation></author><author><name sortKey="Chamova, Teodora" sort="Chamova, Teodora" uniqKey="Chamova T" first="Teodora" last="Chamova">Teodora Chamova</name><affiliation wicri:level="3"><nlm:affiliation>Department of Neurology, Medical University-Sofia, Sofia, Bulgaria.</nlm:affiliation><country xml:lang="fr">Bulgarie</country><wicri:regionArea>Department of Neurology, Medical University-Sofia, Sofia</wicri:regionArea><placeName><settlement type="city">Sofia</settlement><region nuts="2">Sofia-ville (oblast)</region></placeName></affiliation></author><author><name sortKey="Guergueltcheva, Velina" sort="Guergueltcheva, Velina" uniqKey="Guergueltcheva V" first="Velina" last="Guergueltcheva">Velina Guergueltcheva</name><affiliation wicri:level="3"><nlm:affiliation>Clinic of Neurology, University Hospital Sofiamed, Sofia, Bulgaria.</nlm:affiliation><country xml:lang="fr">Bulgarie</country><wicri:regionArea>Clinic of Neurology, University Hospital Sofiamed, Sofia</wicri:regionArea><placeName><settlement type="city">Sofia</settlement><region nuts="2">Sofia-ville (oblast)</region></placeName></affiliation></author><author><name sortKey="Chan, Sophelia" sort="Chan, Sophelia" uniqKey="Chan S" first="Sophelia" last="Chan">Sophelia Chan</name><affiliation><nlm:affiliation>Department of Paediatrics and Adolescent Medicine, Queen Mary Hospital, University of Hong Kong.</nlm:affiliation><wicri:noCountry code="subField">University of Hong Kong</wicri:noCountry></affiliation></author><author><name sortKey="Korngut, Lawrence" sort="Korngut, Lawrence" uniqKey="Korngut L" first="Lawrence" last="Korngut">Lawrence Korngut</name><affiliation wicri:level="4"><nlm:affiliation>Department of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, South Health Campus, Calgary, AB, Canada.</nlm:affiliation><country xml:lang="fr">Canada</country><wicri:regionArea>Department of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, South Health Campus, Calgary, AB</wicri:regionArea><orgName type="university">Université de Calgary</orgName><placeName><settlement type="city">Calgary</settlement><region type="state">Alberta</region></placeName></affiliation></author><author><name sortKey="Campbell, Craig" sort="Campbell, Craig" uniqKey="Campbell C" first="Craig" last="Campbell">Craig Campbell</name><affiliation wicri:level="1"><nlm:affiliation>Department of Paediatrics, Clinical Neurological Sciences & Epidemiology, Western University, London, ON, Canada.</nlm:affiliation><country xml:lang="fr">Canada</country><wicri:regionArea>Department of Paediatrics, Clinical Neurological Sciences & Epidemiology, Western University, London, ON</wicri:regionArea><wicri:noRegion>ON</wicri:noRegion></affiliation></author><author><name sortKey="Dai, Yi" sort="Dai, Yi" uniqKey="Dai Y" first="Yi" last="Dai">Yi Dai</name><affiliation wicri:level="3"><nlm:affiliation>Department of Neurology, Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, Beijing, China.</nlm:affiliation><country xml:lang="fr">République populaire de Chine</country><wicri:regionArea>Department of Neurology, Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, Beijing</wicri:regionArea><placeName><settlement type="city">Pékin</settlement></placeName></affiliation></author><author><name sortKey="Wang, Jen" sort="Wang, Jen" uniqKey="Wang J" first="Jen" last="Wang">Jen Wang</name><affiliation wicri:level="1"><nlm:affiliation>China DMD Care and Support Association c/o China Dolls, Xicheng district, China.</nlm:affiliation><country xml:lang="fr">République populaire de Chine</country><wicri:regionArea>China DMD Care and Support Association c/o China Dolls, Xicheng district</wicri:regionArea><wicri:noRegion>Xicheng district</wicri:noRegion></affiliation></author><author><name sortKey="Barisi, Nina" sort="Barisi, Nina" uniqKey="Barisi N" first="Nina" last="Bariši">Nina Bariši</name><affiliation wicri:level="1"><nlm:affiliation>Division of Paediatric Neurology, University Hospital Centre Zagreb (KBC Zagreb) University of Zagreb Medical School, Croatia.</nlm:affiliation><country xml:lang="fr">Croatie</country><wicri:regionArea>Division of Paediatric Neurology, University Hospital Centre Zagreb (KBC Zagreb) University of Zagreb Medical School</wicri:regionArea><wicri:noRegion>University Hospital Centre Zagreb (KBC Zagreb) University of Zagreb Medical School</wicri:noRegion></affiliation></author><author><name sortKey="Brabec, Petr" sort="Brabec, Petr" uniqKey="Brabec P" first="Petr" last="Brabec">Petr Brabec</name><affiliation wicri:level="3"><nlm:affiliation>Institute for Biostatistic and Analyses, Masaryk University, Brno, Czech Republic.</nlm:affiliation><country xml:lang="fr">République tchèque</country><wicri:regionArea>Institute for Biostatistic and Analyses, Masaryk University, Brno</wicri:regionArea><placeName><settlement type="city">Brno</settlement><region>Moravie</region></placeName></affiliation></author><author><name sortKey="L Hdetie, Jaana" sort="L Hdetie, Jaana" uniqKey="L Hdetie J" first="Jaana" last="L Hdetie">Jaana L Hdetie</name><affiliation wicri:level="3"><nlm:affiliation>Department of Child Neurology, Turku University Central Hospital, Turku, Finland.</nlm:affiliation><country xml:lang="fr">Finlande</country><wicri:regionArea>Department of Child Neurology, Turku University Central Hospital, Turku</wicri:regionArea><placeName><settlement type="city">Turku</settlement><region type="région" nuts="2">Finlande occidentale</region></placeName></affiliation></author><author><name sortKey="Walter, Maggie C" sort="Walter, Maggie C" uniqKey="Walter M" first="Maggie C" last="Walter">Maggie C. Walter</name><affiliation wicri:level="4"><nlm:affiliation>Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany.</nlm:affiliation><country xml:lang="fr">Allemagne</country><wicri:regionArea>Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich</wicri:regionArea><placeName><region type="land" nuts="1">Bavière</region><region type="district" nuts="2">District de Haute-Bavière</region><settlement type="city">Munich</settlement><settlement type="city">Munich</settlement></placeName><orgName type="university">Université Louis-et-Maximilien de Munich</orgName></affiliation></author><author><name sortKey="Schreiber Katz, Olivia" sort="Schreiber Katz, Olivia" uniqKey="Schreiber Katz O" first="Olivia" last="Schreiber-Katz">Olivia Schreiber-Katz</name><affiliation wicri:level="4"><nlm:affiliation>Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany.</nlm:affiliation><country xml:lang="fr">Allemagne</country><wicri:regionArea>Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich</wicri:regionArea><placeName><region type="land" nuts="1">Bavière</region><region type="district" nuts="2">District de Haute-Bavière</region><settlement type="city">Munich</settlement><settlement type="city">Munich</settlement></placeName><orgName type="university">Université Louis-et-Maximilien de Munich</orgName></affiliation></author><author><name sortKey="Karcagi, Veronika" sort="Karcagi, Veronika" uniqKey="Karcagi V" first="Veronika" last="Karcagi">Veronika Karcagi</name><affiliation wicri:level="3"><nlm:affiliation>NIEH, Department of Molecular Genetics and Diagnostics Albert Florian str 2-6, Budapest, Hungary.</nlm:affiliation><country xml:lang="fr">Hongrie</country><wicri:regionArea>NIEH, Department of Molecular Genetics and Diagnostics Albert Florian str 2-6, Budapest</wicri:regionArea><placeName><settlement type="city">Budapest</settlement><region nuts="2">Hongrie centrale</region></placeName></affiliation></author><author><name sortKey="Garami, Marta" sort="Garami, Marta" uniqKey="Garami M" first="Marta" last="Garami">Marta Garami</name><affiliation wicri:level="3"><nlm:affiliation>NIEH, Department of Molecular Genetics and Diagnostics Albert Florian str 2-6, Budapest, Hungary.</nlm:affiliation><country xml:lang="fr">Hongrie</country><wicri:regionArea>NIEH, Department of Molecular Genetics and Diagnostics Albert Florian str 2-6, Budapest</wicri:regionArea><placeName><settlement type="city">Budapest</settlement><region nuts="2">Hongrie centrale</region></placeName></affiliation></author><author><name sortKey="Herczegfalvi, Agnes" sort="Herczegfalvi, Agnes" uniqKey="Herczegfalvi A" first="Agnes" last="Herczegfalvi">Agnes Herczegfalvi</name><affiliation wicri:level="1"><nlm:affiliation>Semmelweis Medical University, II. Department of Paediatric Neurology, Budapest, Tűzoltó u, Hungary.</nlm:affiliation><country xml:lang="fr">Hongrie</country><wicri:regionArea>Semmelweis Medical University, II. Department of Paediatric Neurology, Budapest, Tűzoltó u</wicri:regionArea><wicri:noRegion>Tűzoltó u</wicri:noRegion></affiliation></author><author><name sortKey="Viswanathan, Venkatarman" sort="Viswanathan, Venkatarman" uniqKey="Viswanathan V" first="Venkatarman" last="Viswanathan">Venkatarman Viswanathan</name><affiliation wicri:level="1"><nlm:affiliation>Kanchi Kamakoti CHILDS Trust and Apollo Children's Hospitals, Chennai, India.</nlm:affiliation><country xml:lang="fr">Inde</country><wicri:regionArea>Kanchi Kamakoti CHILDS Trust and Apollo Children's Hospitals, Chennai</wicri:regionArea><wicri:noRegion>Chennai</wicri:noRegion></affiliation></author><author><name sortKey="Bayat, Farhad" sort="Bayat, Farhad" uniqKey="Bayat F" first="Farhad" last="Bayat">Farhad Bayat</name><affiliation wicri:level="3"><nlm:affiliation>Pasteur Institute of Iran, Karaj complex, Tehran, Iran. Parent Project Onlus, Roma, Italy.</nlm:affiliation><country xml:lang="fr">Italie</country><wicri:regionArea>Pasteur Institute of Iran, Karaj complex, Tehran, Iran. Parent Project Onlus, Roma</wicri:regionArea><placeName><settlement type="city">Rome</settlement><region nuts="2">Latium</region></placeName></affiliation></author><author><name sortKey="Buccella, Filippo" sort="Buccella, Filippo" uniqKey="Buccella F" first="Filippo" last="Buccella">Filippo Buccella</name><affiliation wicri:level="3"><nlm:affiliation>Parent Project Onlus, Roma, Italy.</nlm:affiliation><country xml:lang="fr">Italie</country><wicri:regionArea>Parent Project Onlus, Roma</wicri:regionArea><placeName><settlement type="city">Rome</settlement><region nuts="2">Latium</region></placeName></affiliation></author><author><name sortKey="Ferlini, Alessandra" sort="Ferlini, Alessandra" uniqKey="Ferlini A" first="Alessandra" last="Ferlini">Alessandra Ferlini</name><affiliation wicri:level="1"><nlm:affiliation>Department of Reproduction and Growth, Department of Medical Sciences, OSPFE, University of Ferrara, Italy.</nlm:affiliation><country xml:lang="fr">Italie</country><wicri:regionArea>Department of Reproduction and Growth, Department of Medical Sciences, OSPFE, University of Ferrara</wicri:regionArea><wicri:noRegion>University of Ferrara</wicri:noRegion></affiliation></author><author><name sortKey="Kimura, En" sort="Kimura, En" uniqKey="Kimura E" first="En" last="Kimura">En Kimura</name><affiliation wicri:level="3"><nlm:affiliation>214-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.</nlm:affiliation><country xml:lang="fr">Japon</country><wicri:regionArea>214-1-1 Ogawa-Higashi, Kodaira, Tokyo</wicri:regionArea><placeName><settlement type="city">Tokyo</settlement><region type="région">Région de Kantō</region></placeName></affiliation></author><author><name sortKey="Van Den Bergen, Janneke C" sort="Van Den Bergen, Janneke C" uniqKey="Van Den Bergen J" first="Janneke C" last="Van Den Bergen">Janneke C. Van Den Bergen</name><affiliation wicri:level="1"><nlm:affiliation>Leiden University Medical Center, Department of Neurology, ZA Leiden, The Netherlands.</nlm:affiliation><country xml:lang="fr">Pays-Bas</country><wicri:regionArea>Leiden University Medical Center, Department of Neurology, ZA Leiden</wicri:regionArea><wicri:noRegion>ZA Leiden</wicri:noRegion></affiliation></author><author><name sortKey="Rodrigues, Miriam" sort="Rodrigues, Miriam" uniqKey="Rodrigues M" first="Miriam" last="Rodrigues">Miriam Rodrigues</name><affiliation wicri:level="1"><nlm:affiliation>Department of Neurology, Auckland DHB, Auckland, New Zealand.</nlm:affiliation><country xml:lang="fr">Nouvelle-Zélande</country><wicri:regionArea>Department of Neurology, Auckland DHB, Auckland</wicri:regionArea><wicri:noRegion>Auckland</wicri:noRegion></affiliation></author><author><name sortKey="Roxburgh, Richard" sort="Roxburgh, Richard" uniqKey="Roxburgh R" first="Richard" last="Roxburgh">Richard Roxburgh</name><affiliation wicri:level="1"><nlm:affiliation>Department of Neurology, Auckland DHB, Auckland, New Zealand.</nlm:affiliation><country xml:lang="fr">Nouvelle-Zélande</country><wicri:regionArea>Department of Neurology, Auckland DHB, Auckland</wicri:regionArea><wicri:noRegion>Auckland</wicri:noRegion></affiliation></author><author><name sortKey="Lusakowska, Anna" sort="Lusakowska, Anna" uniqKey="Lusakowska A" first="Anna" last="Lusakowska">Anna Lusakowska</name><affiliation><nlm:affiliation>Department of Neurology, Medical University of Warsaw, Warsaw. Poland.</nlm:affiliation><wicri:noCountry code="subField">Warsaw</wicri:noCountry></affiliation></author><author><name sortKey="Kostera Pruszczyk, Anna" sort="Kostera Pruszczyk, Anna" uniqKey="Kostera Pruszczyk A" first="Anna" last="Kostera-Pruszczyk">Anna Kostera-Pruszczyk</name><affiliation><nlm:affiliation>Department of Neurology, Medical University of Warsaw, Warsaw. Poland.</nlm:affiliation><wicri:noCountry code="subField">Warsaw</wicri:noCountry></affiliation></author><author><name sortKey="Santos, Rosario" sort="Santos, Rosario" uniqKey="Santos R" first="Rosário" last="Santos">Rosário Santos</name><affiliation wicri:level="1"><nlm:affiliation>Centro de Genética Médica Jacinto Magalhães, Porto, Portugal.</nlm:affiliation><country xml:lang="fr">Portugal</country><wicri:regionArea>Centro de Genética Médica Jacinto Magalhães, Porto</wicri:regionArea><wicri:noRegion>Porto</wicri:noRegion></affiliation></author><author><name sortKey="Neagu, Elena" sort="Neagu, Elena" uniqKey="Neagu E" first="Elena" last="Neagu">Elena Neagu</name><affiliation wicri:level="1"><nlm:affiliation>National Institute of Legal Medicine "Mina Minovici" - Genetics Laboratory, Bucharest, Romania.</nlm:affiliation><country xml:lang="fr">Roumanie</country><wicri:regionArea>National Institute of Legal Medicine "Mina Minovici" - Genetics Laboratory, Bucharest</wicri:regionArea><wicri:noRegion>Bucharest</wicri:noRegion></affiliation></author><author><name sortKey="Artemieva, Svetlana" sort="Artemieva, Svetlana" uniqKey="Artemieva S" first="Svetlana" last="Artemieva">Svetlana Artemieva</name><affiliation wicri:level="3"><nlm:affiliation>Moscow Institute of Pediatrics, Moscow, Russia.</nlm:affiliation><country xml:lang="fr">Russie</country><wicri:regionArea>Moscow Institute of Pediatrics, Moscow</wicri:regionArea><placeName><settlement type="city">Moscou</settlement><region>District fédéral central</region></placeName></affiliation></author><author><name sortKey="Rasic, Vedrana Milic" sort="Rasic, Vedrana Milic" uniqKey="Rasic V" first="Vedrana Milic" last="Rasic">Vedrana Milic Rasic</name><affiliation wicri:level="1"><nlm:affiliation>Clinic for Neurology and Psychiatry for Children and Youth, Faculty of Medicine, University of Belgrade, Belgrade, Serbia.</nlm:affiliation><country xml:lang="fr">Serbie</country><wicri:regionArea>Clinic for Neurology and Psychiatry for Children and Youth, Faculty of Medicine, University of Belgrade, Belgrade</wicri:regionArea><wicri:noRegion>Belgrade</wicri:noRegion></affiliation></author><author><name sortKey="Vojinovic, Dina" sort="Vojinovic, Dina" uniqKey="Vojinovic D" first="Dina" last="Vojinovic">Dina Vojinovic</name><affiliation wicri:level="1"><nlm:affiliation>Clinic for Neurology and Psychiatry for Children and Youth, Faculty of Medicine, University of Belgrade, Belgrade, Serbia.</nlm:affiliation><country xml:lang="fr">Serbie</country><wicri:regionArea>Clinic for Neurology and Psychiatry for Children and Youth, Faculty of Medicine, University of Belgrade, Belgrade</wicri:regionArea><wicri:noRegion>Belgrade</wicri:noRegion></affiliation></author><author><name sortKey="Posada, Manuel" sort="Posada, Manuel" uniqKey="Posada M" first="Manuel" last="Posada">Manuel Posada</name><affiliation wicri:level="3"><nlm:affiliation>Institute of Rare Diseases Research, SpainRDR and CIBERER, Institute of Health Carlos III, Madrid, Spain.</nlm:affiliation><country xml:lang="fr">Espagne</country><wicri:regionArea>Institute of Rare Diseases Research, SpainRDR and CIBERER, Institute of Health Carlos III, Madrid</wicri:regionArea><placeName><settlement type="city">Madrid</settlement><region nuts="2" type="region">Communauté de Madrid</region></placeName></affiliation></author><author><name sortKey="Bloetzer, Clemens" sort="Bloetzer, Clemens" uniqKey="Bloetzer C" first="Clemens" last="Bloetzer">Clemens Bloetzer</name><affiliation wicri:level="4"><nlm:affiliation>Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.</nlm:affiliation><country xml:lang="fr">Suisse</country><wicri:regionArea>Institute of Social and Preventive Medicine, University of Bern, Bern</wicri:regionArea><placeName><settlement type="city">Berne</settlement><region type="région" nuts="3">Canton de Berne</region><settlement type="city">Berne</settlement></placeName><orgName type="university">Université de Berne</orgName></affiliation></author><author><name sortKey="Klein, Andrea" sort="Klein, Andrea" uniqKey="Klein A" first="Andrea" last="Klein">Andrea Klein</name><affiliation wicri:level="3"><nlm:affiliation>Paediatric Neurology and Neurorehabilitation Unit, Lausanne University Hospital, Lausanne, Switzerland.</nlm:affiliation><country xml:lang="fr">Suisse</country><wicri:regionArea>Paediatric Neurology and Neurorehabilitation Unit, Lausanne University Hospital, Lausanne</wicri:regionArea><placeName><settlement type="city">Lausanne</settlement><region nuts="3" type="region">Canton de Vaud</region></placeName></affiliation></author><author><name sortKey="Diaz Manera, Jordi" sort="Diaz Manera, Jordi" uniqKey="Diaz Manera J" first="Jordi" last="Díaz-Manera">Jordi Díaz-Manera</name><affiliation><nlm:affiliation>Unitat de Malalties Neuromusculars, Servei de Neurologia, Hospital de la Santa Creu i Sant Pau de Barcelona.</nlm:affiliation><wicri:noCountry code="subField">Hospital de la Santa Creu i Sant Pau de Barcelona</wicri:noCountry></affiliation></author><author><name sortKey="Gallardo, Eduard" sort="Gallardo, Eduard" uniqKey="Gallardo E" first="Eduard" last="Gallardo">Eduard Gallardo</name><affiliation wicri:level="3"><nlm:affiliation>Paediatric Neurology and Neurorehabilitation Unit, Lausanne University Hospital, Lausanne, Switzerland.</nlm:affiliation><country xml:lang="fr">Suisse</country><wicri:regionArea>Paediatric Neurology and Neurorehabilitation Unit, Lausanne University Hospital, Lausanne</wicri:regionArea><placeName><settlement type="city">Lausanne</settlement><region nuts="3" type="region">Canton de Vaud</region></placeName></affiliation></author><author><name sortKey="Karaduman, A Ay E" sort="Karaduman, A Ay E" uniqKey="Karaduman A" first="A Ay E" last="Karaduman">A Ay E Karaduman</name><affiliation wicri:level="1"><nlm:affiliation>Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara, Turkey.</nlm:affiliation><country xml:lang="fr">Turquie</country><wicri:regionArea>Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara</wicri:regionArea><wicri:noRegion>Ankara</wicri:noRegion></affiliation></author><author><name sortKey="Oznur, Tunca" sort="Oznur, Tunca" uniqKey="Oznur T" first="Tunca" last="Oznur">Tunca Oznur</name><affiliation wicri:level="1"><nlm:affiliation>Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara, Turkey.</nlm:affiliation><country xml:lang="fr">Turquie</country><wicri:regionArea>Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara</wicri:regionArea><wicri:noRegion>Ankara</wicri:noRegion></affiliation></author><author><name sortKey="Topalo Lu, Haluk" sort="Topalo Lu, Haluk" uniqKey="Topalo Lu H" first="Haluk" last="Topalo Lu">Haluk Topalo Lu</name><affiliation wicri:level="1"><nlm:affiliation>Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara, Turkey.</nlm:affiliation><country xml:lang="fr">Turquie</country><wicri:regionArea>Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara</wicri:regionArea><wicri:noRegion>Ankara</wicri:noRegion></affiliation></author><author><name sortKey="El Sherif, Rasha" sort="El Sherif, Rasha" uniqKey="El Sherif R" first="Rasha" last="El Sherif">Rasha El Sherif</name><affiliation wicri:level="1"><nlm:affiliation>Neurology & Neurogenic Unit, Egypt Air Hospital, Ain Shams University, Egypt.</nlm:affiliation><country xml:lang="fr">Égypte</country><wicri:regionArea>Neurology & Neurogenic Unit, Egypt Air Hospital, Ain Shams University</wicri:regionArea><wicri:noRegion>Ain Shams University</wicri:noRegion></affiliation></author><author><name sortKey="Stringer, Angela" sort="Stringer, Angela" uniqKey="Stringer A" first="Angela" last="Stringer">Angela Stringer</name><affiliation wicri:level="3"><nlm:affiliation>Action Duchenne, Epicentre, London, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Action Duchenne, Epicentre, London</wicri:regionArea><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region></placeName></affiliation></author><author><name sortKey="Shatillo, Andriy V" sort="Shatillo, Andriy V" uniqKey="Shatillo A" first="Andriy V" last="Shatillo">Andriy V. Shatillo</name><affiliation wicri:level="1"><nlm:affiliation>Institute of Neurology, Psychiatry and Narcology of NAMS, Kharkiv, Ukraine.</nlm:affiliation><country xml:lang="fr">Ukraine</country><wicri:regionArea>Institute of Neurology, Psychiatry and Narcology of NAMS, Kharkiv</wicri:regionArea><wicri:noRegion>Kharkiv</wicri:noRegion></affiliation></author><author><name sortKey="Martin, Ann S" sort="Martin, Ann S" uniqKey="Martin A" first="Ann S" last="Martin">Ann S. Martin</name><affiliation wicri:level="2"><nlm:affiliation>DuchenneConnect, Hackensack, NJ, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>DuchenneConnect, Hackensack, NJ</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="Peay, Holly L" sort="Peay, Holly L" uniqKey="Peay H" first="Holly L" last="Peay">Holly L. Peay</name><affiliation wicri:level="2"><nlm:affiliation>DuchenneConnect, Hackensack, NJ, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>DuchenneConnect, Hackensack, NJ</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="Kirschner, Jan" sort="Kirschner, Jan" uniqKey="Kirschner J" first="Jan" last="Kirschner">Jan Kirschner</name><affiliation wicri:level="1"><nlm:affiliation>University Medical Center Freiburg, Germany.</nlm:affiliation><country xml:lang="fr">Allemagne</country><wicri:regionArea>University Medical Center Freiburg</wicri:regionArea></affiliation></author><author><name sortKey="Flanigan, Kevin M" sort="Flanigan, Kevin M" uniqKey="Flanigan K" first="Kevin M" last="Flanigan">Kevin M. Flanigan</name><affiliation wicri:level="2"><nlm:affiliation>Center for Gene Therapy, The Research Institute, Nationwide Children's Hospital, Columbus, OH, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>Center for Gene Therapy, The Research Institute, Nationwide Children's Hospital, Columbus, OH</wicri:regionArea><placeName><region type="state">Ohio</region></placeName></affiliation></author><author><name sortKey="Straub, Volker" sort="Straub, Volker" uniqKey="Straub V" first="Volker" last="Straub">Volker Straub</name><affiliation wicri:level="1"><nlm:affiliation>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne</wicri:regionArea><wicri:noRegion>Newcastle upon Tyne</wicri:noRegion></affiliation></author><author><name sortKey="Bushby, Kate" sort="Bushby, Kate" uniqKey="Bushby K" first="Kate" last="Bushby">Kate Bushby</name><affiliation wicri:level="1"><nlm:affiliation>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne</wicri:regionArea><wicri:noRegion>Newcastle upon Tyne</wicri:noRegion></affiliation></author><author><name sortKey="Beroud, Christophe" sort="Beroud, Christophe" uniqKey="Beroud C" first="Christophe" last="Béroud">Christophe Béroud</name><affiliation wicri:level="3"><nlm:affiliation>AP-HM, Hôpital d'Enfants de la Timone, Département de Génétique Médicale et de Biologie Cellulaire, Marseille, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>AP-HM, Hôpital d'Enfants de la Timone, Département de Génétique Médicale et de Biologie Cellulaire, Marseille</wicri:regionArea><placeName><region type="region">Provence-Alpes-Côte d'Azur</region><region type="old region">Provence-Alpes-Côte d'Azur</region><settlement type="city">Marseille</settlement></placeName></affiliation></author><author><name sortKey="Verschuuren, Jan J" sort="Verschuuren, Jan J" uniqKey="Verschuuren J" first="Jan J" last="Verschuuren">Jan J. Verschuuren</name><affiliation wicri:level="1"><nlm:affiliation>Leiden University Medical Center, Department of Neurology, ZA Leiden, The Netherlands.</nlm:affiliation><country xml:lang="fr">Pays-Bas</country><wicri:regionArea>Leiden University Medical Center, Department of Neurology, ZA Leiden</wicri:regionArea><wicri:noRegion>ZA Leiden</wicri:noRegion></affiliation></author><author><name sortKey="Lochmuller, Hanns" sort="Lochmuller, Hanns" uniqKey="Lochmuller H" first="Hanns" last="Lochmüller">Hanns Lochmüller</name><affiliation wicri:level="1"><nlm:affiliation>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne</wicri:regionArea><wicri:noRegion>Newcastle upon Tyne</wicri:noRegion></affiliation></author></analytic><series><title level="j">Journal of neuromuscular diseases</title><idno type="ISSN">2214-3599</idno><imprint><date when="2017" type="published">2017</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Recent short-term clinical trials in patients with Duchenne Muscular Dystrophy (DMD) have indicated greater disease variability in terms of progression than expected. In addition, as average life-expectancy increases, reliable data is required on clinical progression in the older DMD population.</div></front></TEI><affiliations><list><country><li>Allemagne</li><li>Argentine</li><li>Australie</li><li>Belgique</li><li>Bulgarie</li><li>Canada</li><li>Croatie</li><li>Espagne</li><li>Finlande</li><li>France</li><li>Grèce</li><li>Hongrie</li><li>Inde</li><li>Italie</li><li>Japon</li><li>Nouvelle-Zélande</li><li>Pays-Bas</li><li>Portugal</li><li>Roumanie</li><li>Royaume-Uni</li><li>Russie</li><li>République populaire de Chine</li><li>République tchèque</li><li>Serbie</li><li>Suisse</li><li>Turquie</li><li>Ukraine</li><li>Égypte</li><li>États-Unis</li></country><region><li>Alberta</li><li>Angleterre</li><li>Attique (région)</li><li>Bavière</li><li>Canton de Berne</li><li>Canton de Vaud</li><li>Communauté de Madrid</li><li>District de Haute-Bavière</li><li>District fédéral central</li><li>Finlande occidentale</li><li>Grand Londres</li><li>Hongrie centrale</li><li>Latium</li><li>Moravie</li><li>New Jersey</li><li>Ohio</li><li>Provence-Alpes-Côte d'Azur</li><li>Région de Bruxelles-Capitale</li><li>Région de Kantō</li><li>Sofia-ville (oblast)</li></region><settlement><li>Athènes</li><li>Berne</li><li>Brno</li><li>Bruxelles</li><li>Budapest</li><li>Calgary</li><li>Lausanne</li><li>Londres</li><li>Madrid</li><li>Marseille</li><li>Moscou</li><li>Munich</li><li>Pékin</li><li>Rome</li><li>Sofia</li><li>Tokyo</li><li>Turku</li></settlement><orgName><li>Université Louis-et-Maximilien de Munich</li><li>Université de Berne</li><li>Université de Calgary</li></orgName></list><tree><noCountry><name sortKey="Chan, Sophelia" sort="Chan, Sophelia" uniqKey="Chan S" first="Sophelia" last="Chan">Sophelia Chan</name><name sortKey="Diaz Manera, Jordi" sort="Diaz Manera, Jordi" uniqKey="Diaz Manera J" first="Jordi" last="Díaz-Manera">Jordi Díaz-Manera</name><name sortKey="Kostera Pruszczyk, Anna" sort="Kostera Pruszczyk, Anna" uniqKey="Kostera Pruszczyk A" first="Anna" last="Kostera-Pruszczyk">Anna Kostera-Pruszczyk</name><name sortKey="Lusakowska, Anna" sort="Lusakowska, Anna" uniqKey="Lusakowska A" first="Anna" last="Lusakowska">Anna Lusakowska</name></noCountry><country name="Pays-Bas"><noRegion><name sortKey="Koeks, Zaida" sort="Koeks, Zaida" uniqKey="Koeks Z" first="Zaïda" last="Koeks">Zaïda Koeks</name></noRegion><name sortKey="Van Den Bergen, Janneke C" sort="Van Den Bergen, Janneke C" uniqKey="Van Den Bergen J" first="Janneke C" last="Van Den Bergen">Janneke C. Van Den Bergen</name><name sortKey="Van Zwet, Erik" sort="Van Zwet, Erik" uniqKey="Van Zwet E" first="Erik" last="Van Zwet">Erik Van Zwet</name><name sortKey="Verschuuren, Jan J" sort="Verschuuren, Jan J" uniqKey="Verschuuren J" first="Jan J" last="Verschuuren">Jan J. Verschuuren</name></country><country name="Royaume-Uni"><noRegion><name sortKey="Bladen, Catherine L" sort="Bladen, Catherine L" uniqKey="Bladen C" first="Catherine L" last="Bladen">Catherine L. Bladen</name></noRegion><name sortKey="Bushby, Kate" sort="Bushby, Kate" uniqKey="Bushby K" first="Kate" last="Bushby">Kate Bushby</name><name sortKey="Lochmuller, Hanns" sort="Lochmuller, Hanns" uniqKey="Lochmuller H" first="Hanns" last="Lochmüller">Hanns Lochmüller</name><name sortKey="Mcmacken, Grace" sort="Mcmacken, Grace" uniqKey="Mcmacken G" first="Grace" last="Mcmacken">Grace Mcmacken</name><name sortKey="Pogoryelova, Oksana" sort="Pogoryelova, Oksana" uniqKey="Pogoryelova O" first="Oksana" last="Pogoryelova">Oksana Pogoryelova</name><name sortKey="Straub, Volker" sort="Straub, Volker" uniqKey="Straub V" first="Volker" last="Straub">Volker Straub</name><name sortKey="Stringer, Angela" sort="Stringer, Angela" uniqKey="Stringer A" first="Angela" last="Stringer">Angela Stringer</name></country><country name="France"><region name="Provence-Alpes-Côte d'Azur"><name sortKey="Salgado, David" sort="Salgado, David" uniqKey="Salgado D" first="David" last="Salgado">David Salgado</name></region><name sortKey="Beroud, Christophe" sort="Beroud, Christophe" uniqKey="Beroud C" first="Christophe" last="Béroud">Christophe Béroud</name></country><country name="Argentine"><noRegion><name sortKey="Monges, Soledad" sort="Monges, Soledad" uniqKey="Monges S" first="Soledad" last="Monges">Soledad Monges</name></noRegion><name sortKey="Foncuberta, Maria" sort="Foncuberta, Maria" uniqKey="Foncuberta M" first="Maria" last="Foncuberta">Maria Foncuberta</name></country><country name="Grèce"><region name="Attique (région)"><name sortKey="Kekou, Kyriaki" sort="Kekou, Kyriaki" uniqKey="Kekou K" first="Kyriaki" last="Kekou">Kyriaki Kekou</name></region><name sortKey="Kosma, Konstantina" sort="Kosma, Konstantina" uniqKey="Kosma K" first="Konstantina" last="Kosma">Konstantina Kosma</name></country><country name="Australie"><noRegion><name sortKey="Dawkins, Hugh" sort="Dawkins, Hugh" uniqKey="Dawkins H" first="Hugh" last="Dawkins">Hugh Dawkins</name></noRegion><name sortKey="Bellgard, Matthew I" sort="Bellgard, Matthew I" uniqKey="Bellgard M" first="Matthew I" last="Bellgard">Matthew I. 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Shatillo</name></noRegion></country><country name="États-Unis"><region name="New Jersey"><name sortKey="Martin, Ann S" sort="Martin, Ann S" uniqKey="Martin A" first="Ann S" last="Martin">Ann S. Martin</name></region><name sortKey="Flanigan, Kevin M" sort="Flanigan, Kevin M" uniqKey="Flanigan K" first="Kevin M" last="Flanigan">Kevin M. Flanigan</name><name sortKey="Peay, Holly L" sort="Peay, Holly L" uniqKey="Peay H" first="Holly L" last="Peay">Holly L. Peay</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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