Ataluren treatment of patients with nonsense mutation dystrophinopathy
Identifieur interne : 001C39 ( Ncbi/Curation ); précédent : 001C38; suivant : 001C40Ataluren treatment of patients with nonsense mutation dystrophinopathy
Auteurs : Katharine Bushby [Royaume-Uni] ; Richard Finkel [États-Unis] ; Brenda Wong [États-Unis] ; Richard Barohn [États-Unis] ; Craig Campbell [Canada] ; Giacomo P. Comi [Italie] ; Anne M. Connolly [États-Unis] ; John W. Day [États-Unis] ; Kevin M. Flanigan [États-Unis] ; Nathalie Goemans [Belgique] ; Kristi J. Jones ; Eugenio Mercuri [Italie] ; Ros Quinlivan [Royaume-Uni] ; James B. Renfroe [États-Unis] ; Barry Russman [États-Unis] ; Monique M. Ryan [Australie] ; Mar Tulinius [Suède] ; Thomas Voit [France] ; Steven A. Moore [États-Unis] ; H. Lee Sweeney [États-Unis] ; Richard T. Abresch [États-Unis] ; Kim L. Coleman [États-Unis] ; Michelle Eagle [Royaume-Uni] ; Julaine Florence [États-Unis] ; Eduard Gappmaier [États-Unis] ; Allan M. Glanzman [États-Unis] ; Erik Henricson [États-Unis] ; Jay Barth [États-Unis] ; Gary L. Elfring [États-Unis] ; Allen Reha [États-Unis] ; Robert J. Spiegel [États-Unis] ; Michael W. O'Donnell [États-Unis] ; Stuart W. Peltz [États-Unis] ; Craig M. Mcdonald [États-Unis]Source :
- Muscle & Nerve [ 0148-639X ] ; 2014.
Abstract
Url:
DOI: 10.1002/mus.24332
PubMed: 25042182
PubMed Central: 4241581
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PMC:4241581Le document en format XML
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Moore</name><affiliation wicri:level="1"><nlm:aff id="au19"><institution>University of Iowa</institution><addr-line>Iowa, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Iowa</wicri:regionArea></affiliation></author><author><name sortKey="Lee Sweeney, H" sort="Lee Sweeney, H" uniqKey="Lee Sweeney H" first="H" last="Lee Sweeney">H. Lee Sweeney</name><affiliation wicri:level="1"><nlm:aff id="au20"><institution>University of Pennsylvania</institution><addr-line>Pennsylvania, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Pennsylvania</wicri:regionArea></affiliation></author><author><name sortKey="Abresch, Richard T" sort="Abresch, Richard T" uniqKey="Abresch R" first="Richard T" last="Abresch">Richard T. Abresch</name><affiliation wicri:level="1"><nlm:aff id="au21"><institution>UC Davis Children's Hospital, Lawrence J. Ellison Ambulatory Care Center, Physical Medicine & Rehabilitation</institution><addr-line>4860 Y St., Suite 1700, Sacramento, California, 95817, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>4860 Y St., Suite 1700, Sacramento, California, 95817</wicri:regionArea><wicri:noRegion>95817</wicri:noRegion></affiliation></author><author><name sortKey="Coleman, Kim L" sort="Coleman, Kim L" uniqKey="Coleman K" first="Kim L" last="Coleman">Kim L. 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Glanzman</name><affiliation wicri:level="1"><nlm:aff id="au2"><institution>The Children's Hospital of Philadelphia</institution><addr-line>Pennsylvania, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Pennsylvania</wicri:regionArea></affiliation></author><author><name sortKey="Henricson, Erik" sort="Henricson, Erik" uniqKey="Henricson E" first="Erik" last="Henricson">Erik Henricson</name><affiliation wicri:level="1"><nlm:aff id="au21"><institution>UC Davis Children's Hospital, Lawrence J. Ellison Ambulatory Care Center, Physical Medicine & Rehabilitation</institution><addr-line>4860 Y St., Suite 1700, Sacramento, California, 95817, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>4860 Y St., Suite 1700, Sacramento, California, 95817</wicri:regionArea><wicri:noRegion>95817</wicri:noRegion></affiliation></author><author><name sortKey="Barth, Jay" sort="Barth, Jay" uniqKey="Barth J" first="Jay" last="Barth">Jay Barth</name><affiliation wicri:level="2"><nlm:aff id="au24"><institution>PTC Therapeutics</institution><addr-line>South Plainfield, New Jersey, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>South Plainfield, New Jersey</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="Elfring, Gary L" sort="Elfring, Gary L" uniqKey="Elfring G" first="Gary L" last="Elfring">Gary L. Elfring</name><affiliation wicri:level="2"><nlm:aff id="au24"><institution>PTC Therapeutics</institution><addr-line>South Plainfield, New Jersey, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>South Plainfield, New Jersey</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="Reha, Allen" sort="Reha, Allen" uniqKey="Reha A" first="Allen" last="Reha">Allen Reha</name><affiliation wicri:level="2"><nlm:aff id="au24"><institution>PTC Therapeutics</institution><addr-line>South Plainfield, New Jersey, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>South Plainfield, New Jersey</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="Spiegel, Robert J" sort="Spiegel, Robert J" uniqKey="Spiegel R" first="Robert J" last="Spiegel">Robert J. Spiegel</name><affiliation wicri:level="2"><nlm:aff id="au24"><institution>PTC Therapeutics</institution><addr-line>South Plainfield, New Jersey, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>South Plainfield, New Jersey</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="O Donnell, Michael W" sort="O Donnell, Michael W" uniqKey="O Donnell M" first="Michael W" last="O'Donnell">Michael W. O'Donnell</name><affiliation wicri:level="2"><nlm:aff id="au24"><institution>PTC Therapeutics</institution><addr-line>South Plainfield, New Jersey, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>South Plainfield, New Jersey</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="Peltz, Stuart W" sort="Peltz, Stuart W" uniqKey="Peltz S" first="Stuart W" last="Peltz">Stuart W. Peltz</name><affiliation wicri:level="2"><nlm:aff id="au24"><institution>PTC Therapeutics</institution><addr-line>South Plainfield, New Jersey, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>South Plainfield, New Jersey</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="Mcdonald, Craig M" sort="Mcdonald, Craig M" uniqKey="Mcdonald C" first="Craig M" last="Mcdonald">Craig M. Mcdonald</name><affiliation wicri:level="1"><nlm:aff id="au21"><institution>UC Davis Children's Hospital, Lawrence J. Ellison Ambulatory Care Center, Physical Medicine & Rehabilitation</institution><addr-line>4860 Y St., Suite 1700, Sacramento, California, 95817, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>4860 Y St., Suite 1700, Sacramento, California, 95817</wicri:regionArea><wicri:noRegion>95817</wicri:noRegion></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PMC</idno><idno type="pmid">25042182</idno><idno type="pmc">4241581</idno><idno type="url">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4241581</idno><idno type="RBID">PMC:4241581</idno><idno type="doi">10.1002/mus.24332</idno><date when="2014">2014</date><idno type="wicri:Area/Pmc/Corpus">002778</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Corpus" wicri:corpus="PMC">002778</idno><idno type="wicri:Area/Pmc/Curation">002628</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Curation">002628</idno><idno type="wicri:Area/Pmc/Checkpoint">001737</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Checkpoint">001737</idno><idno type="wicri:Area/Ncbi/Merge">001C39</idno><idno type="wicri:Area/Ncbi/Curation">001C39</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en" level="a" type="main">Ataluren treatment of patients with nonsense mutation dystrophinopathy</title><author><name sortKey="Bushby, Katharine" sort="Bushby, Katharine" uniqKey="Bushby K" first="Katharine" last="Bushby">Katharine Bushby</name><affiliation wicri:level="1"><nlm:aff id="au1"><institution>Institute of Genetic Medicine, Newcastle University</institution><addr-line>Newcastle upon Tyne, United Kingdom</addr-line></nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Newcastle upon Tyne</wicri:regionArea></affiliation></author><author><name sortKey="Finkel, Richard" sort="Finkel, Richard" uniqKey="Finkel R" first="Richard" last="Finkel">Richard Finkel</name><affiliation wicri:level="1"><nlm:aff id="au2"><institution>The Children's Hospital of Philadelphia</institution><addr-line>Pennsylvania, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Pennsylvania</wicri:regionArea></affiliation></author><author><name sortKey="Wong, Brenda" sort="Wong, Brenda" uniqKey="Wong B" first="Brenda" last="Wong">Brenda Wong</name><affiliation wicri:level="1"><nlm:aff id="au3"><institution>Cincinnati Children's Hospital Medical Center</institution><addr-line>Ohio, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Ohio</wicri:regionArea></affiliation></author><author><name sortKey="Barohn, Richard" sort="Barohn, Richard" uniqKey="Barohn R" first="Richard" last="Barohn">Richard Barohn</name><affiliation wicri:level="1"><nlm:aff id="au4"><institution>University of Kansas Medical Center</institution><addr-line>Kansas, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Kansas</wicri:regionArea></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:aff id="au5"><institution>University of Western Ontario</institution><addr-line>London, Ontario, Canada</addr-line></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea>London, Ontario</wicri:regionArea><wicri:noRegion>Ontario</wicri:noRegion></affiliation></author><author><name sortKey="Comi, Giacomo P" sort="Comi, Giacomo P" uniqKey="Comi G" first="Giacomo P" last="Comi">Giacomo P. Comi</name><affiliation wicri:level="3"><nlm:aff id="au6"><institution>Dino Ferrari Centre, Department of Neurological Sciences, University of Milan</institution><addr-line>I.R.C.C.S. Foundation Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy</addr-line></nlm:aff><country xml:lang="fr">Italie</country><wicri:regionArea>I.R.C.C.S. Foundation Ca' Granda, Ospedale Maggiore Policlinico, Milan</wicri:regionArea><placeName><settlement type="city">Milan</settlement><region nuts="2">Lombardie</region></placeName></affiliation></author><author><name sortKey="Connolly, Anne M" sort="Connolly, Anne M" uniqKey="Connolly A" first="Anne M" last="Connolly">Anne M. Connolly</name><affiliation wicri:level="1"><nlm:aff id="au7"><institution>Washington University School of Medicine at St. Louis</institution><addr-line>Missouri, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Missouri</wicri:regionArea></affiliation></author><author><name sortKey="Day, John W" sort="Day, John W" uniqKey="Day J" first="John W" last="Day">John W. Day</name><affiliation wicri:level="2"><nlm:aff id="au8"><institution>University of Minnesota</institution><addr-line>Minneapolis, Minnesota, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Minneapolis, Minnesota</wicri:regionArea><placeName><region type="state">Minnesota</region></placeName></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:aff id="au9"><institution>Nationwide Children's Hospital and the Ohio State University</institution><addr-line>Columbus, Ohio, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Columbus, Ohio</wicri:regionArea><placeName><region type="state">Ohio</region></placeName></affiliation></author><author><name sortKey="Goemans, Nathalie" sort="Goemans, Nathalie" uniqKey="Goemans N" first="Nathalie" last="Goemans">Nathalie Goemans</name><affiliation wicri:level="1"><nlm:aff id="au10"><institution>University Hospitals Leuven</institution><addr-line>Leuven, Belgium</addr-line></nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea>Leuven</wicri:regionArea></affiliation></author><author><name sortKey="Jones, Kristi J" sort="Jones, Kristi J" uniqKey="Jones K" first="Kristi J" last="Jones">Kristi J. Jones</name><affiliation><nlm:aff id="au11"><institution>Department of Clinical Genetics, Sydney Children's Hospital Network, and Disciplines of Genetics and Paediatrics and Child Health, Faculty of Medicine University of Sydney</institution><addr-line>Australia</addr-line></nlm:aff></affiliation></author><author><name sortKey="Mercuri, Eugenio" sort="Mercuri, Eugenio" uniqKey="Mercuri E" first="Eugenio" last="Mercuri">Eugenio Mercuri</name><affiliation wicri:level="1"><nlm:aff id="au12"><institution>Pediatric Neurology Unit, Polilcinico Gemelli, Università Cattolica Sacro Cuore</institution><addr-line>Rome, Italy</addr-line></nlm:aff><country xml:lang="fr">Italie</country><wicri:regionArea>Rome</wicri:regionArea></affiliation></author><author><name sortKey="Quinlivan, Ros" sort="Quinlivan, Ros" uniqKey="Quinlivan R" first="Ros" last="Quinlivan">Ros Quinlivan</name><affiliation wicri:level="1"><nlm:aff id="au13"><institution>The Institute of Neurology</institution><addr-line>London, United Kingdom</addr-line></nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>London</wicri:regionArea></affiliation></author><author><name sortKey="Renfroe, James B" sort="Renfroe, James B" uniqKey="Renfroe J" first="James B" last="Renfroe">James B. Renfroe</name><affiliation wicri:level="1"><nlm:aff id="au14"><institution>Northwest Florida Clinical Research Group</institution><addr-line>Florida, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Florida</wicri:regionArea></affiliation></author><author><name sortKey="Russman, Barry" sort="Russman, Barry" uniqKey="Russman B" first="Barry" last="Russman">Barry Russman</name><affiliation wicri:level="1"><nlm:aff id="au15"><institution>Oregon Health & Science University and Shriners Hospital for Children</institution><addr-line>Oregon, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Oregon</wicri:regionArea></affiliation></author><author><name sortKey="Ryan, Monique M" sort="Ryan, Monique M" uniqKey="Ryan M" first="Monique M" last="Ryan">Monique M. Ryan</name><affiliation wicri:level="1"><nlm:aff id="au16"><institution>Royal Children's Hospital, Murdoch Childrens Research Institute and University of Melbourne</institution><addr-line>Parkville, Victoria, Australia</addr-line></nlm:aff><country xml:lang="fr">Australie</country><wicri:regionArea>Parkville, Victoria</wicri:regionArea><wicri:noRegion>Victoria</wicri:noRegion></affiliation></author><author><name sortKey="Tulinius, Mar" sort="Tulinius, Mar" uniqKey="Tulinius M" first="Mar" last="Tulinius">Mar Tulinius</name><affiliation wicri:level="1"><nlm:aff id="au17"><institution>Department of Pediatrics, The University of Gothenburg</institution><addr-line>Gothenburg, Sweden</addr-line></nlm:aff><country xml:lang="fr">Suède</country><wicri:regionArea>Gothenburg</wicri:regionArea></affiliation></author><author><name sortKey="Voit, Thomas" sort="Voit, Thomas" uniqKey="Voit T" first="Thomas" last="Voit">Thomas Voit</name><affiliation wicri:level="3"><nlm:aff id="au18"><institution>Institut de Myologie, University Pierre et Marie Curie Paris 6</institution><addr-line>UM 76, INSERM U 974, CNRS UMR 7215, Paris, France</addr-line></nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>UM 76, INSERM U 974, CNRS UMR 7215, Paris</wicri:regionArea><placeName><region type="region">Île-de-France</region><region type="old region">Île-de-France</region><settlement type="city">Paris</settlement></placeName></affiliation></author><author><name sortKey="Moore, Steven A" sort="Moore, Steven A" uniqKey="Moore S" first="Steven A" last="Moore">Steven A. Moore</name><affiliation wicri:level="1"><nlm:aff id="au19"><institution>University of Iowa</institution><addr-line>Iowa, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Iowa</wicri:regionArea></affiliation></author><author><name sortKey="Lee Sweeney, H" sort="Lee Sweeney, H" uniqKey="Lee Sweeney H" first="H" last="Lee Sweeney">H. Lee Sweeney</name><affiliation wicri:level="1"><nlm:aff id="au20"><institution>University of Pennsylvania</institution><addr-line>Pennsylvania, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Pennsylvania</wicri:regionArea></affiliation></author><author><name sortKey="Abresch, Richard T" sort="Abresch, Richard T" uniqKey="Abresch R" first="Richard T" last="Abresch">Richard T. Abresch</name><affiliation wicri:level="1"><nlm:aff id="au21"><institution>UC Davis Children's Hospital, Lawrence J. Ellison Ambulatory Care Center, Physical Medicine & Rehabilitation</institution><addr-line>4860 Y St., Suite 1700, Sacramento, California, 95817, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>4860 Y St., Suite 1700, Sacramento, California, 95817</wicri:regionArea><wicri:noRegion>95817</wicri:noRegion></affiliation></author><author><name sortKey="Coleman, Kim L" sort="Coleman, Kim L" uniqKey="Coleman K" first="Kim L" last="Coleman">Kim L. Coleman</name><affiliation wicri:level="2"><nlm:aff id="au22"><institution>OrthoCare Innovations</institution><addr-line>Mountlake Terrace, Washington, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Mountlake Terrace, Washington</wicri:regionArea><placeName><region type="state">Washington (État)</region></placeName></affiliation></author><author><name sortKey="Eagle, Michelle" sort="Eagle, Michelle" uniqKey="Eagle M" first="Michelle" last="Eagle">Michelle Eagle</name><affiliation wicri:level="1"><nlm:aff id="au1"><institution>Institute of Genetic Medicine, Newcastle University</institution><addr-line>Newcastle upon Tyne, United Kingdom</addr-line></nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Newcastle upon Tyne</wicri:regionArea></affiliation></author><author><name sortKey="Florence, Julaine" sort="Florence, Julaine" uniqKey="Florence J" first="Julaine" last="Florence">Julaine Florence</name><affiliation wicri:level="1"><nlm:aff id="au7"><institution>Washington University School of Medicine at St. Louis</institution><addr-line>Missouri, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Missouri</wicri:regionArea></affiliation></author><author><name sortKey="Gappmaier, Eduard" sort="Gappmaier, Eduard" uniqKey="Gappmaier E" first="Eduard" last="Gappmaier">Eduard Gappmaier</name><affiliation wicri:level="2"><nlm:aff id="au23"><institution>University of Utah School of Medicine</institution><addr-line>Salt Lake City, Utah, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Salt Lake City, Utah</wicri:regionArea><placeName><region type="state">Utah</region></placeName></affiliation></author><author><name sortKey="Glanzman, Allan M" sort="Glanzman, Allan M" uniqKey="Glanzman A" first="Allan M" last="Glanzman">Allan M. Glanzman</name><affiliation wicri:level="1"><nlm:aff id="au2"><institution>The Children's Hospital of Philadelphia</institution><addr-line>Pennsylvania, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Pennsylvania</wicri:regionArea></affiliation></author><author><name sortKey="Henricson, Erik" sort="Henricson, Erik" uniqKey="Henricson E" first="Erik" last="Henricson">Erik Henricson</name><affiliation wicri:level="1"><nlm:aff id="au21"><institution>UC Davis Children's Hospital, Lawrence J. Ellison Ambulatory Care Center, Physical Medicine & Rehabilitation</institution><addr-line>4860 Y St., Suite 1700, Sacramento, California, 95817, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>4860 Y St., Suite 1700, Sacramento, California, 95817</wicri:regionArea><wicri:noRegion>95817</wicri:noRegion></affiliation></author><author><name sortKey="Barth, Jay" sort="Barth, Jay" uniqKey="Barth J" first="Jay" last="Barth">Jay Barth</name><affiliation wicri:level="2"><nlm:aff id="au24"><institution>PTC Therapeutics</institution><addr-line>South Plainfield, New Jersey, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>South Plainfield, New Jersey</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="Elfring, Gary L" sort="Elfring, Gary L" uniqKey="Elfring G" first="Gary L" last="Elfring">Gary L. Elfring</name><affiliation wicri:level="2"><nlm:aff id="au24"><institution>PTC Therapeutics</institution><addr-line>South Plainfield, New Jersey, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>South Plainfield, New Jersey</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="Reha, Allen" sort="Reha, Allen" uniqKey="Reha A" first="Allen" last="Reha">Allen Reha</name><affiliation wicri:level="2"><nlm:aff id="au24"><institution>PTC Therapeutics</institution><addr-line>South Plainfield, New Jersey, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>South Plainfield, New Jersey</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="Spiegel, Robert J" sort="Spiegel, Robert J" uniqKey="Spiegel R" first="Robert J" last="Spiegel">Robert J. Spiegel</name><affiliation wicri:level="2"><nlm:aff id="au24"><institution>PTC Therapeutics</institution><addr-line>South Plainfield, New Jersey, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>South Plainfield, New Jersey</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="O Donnell, Michael W" sort="O Donnell, Michael W" uniqKey="O Donnell M" first="Michael W" last="O'Donnell">Michael W. O'Donnell</name><affiliation wicri:level="2"><nlm:aff id="au24"><institution>PTC Therapeutics</institution><addr-line>South Plainfield, New Jersey, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>South Plainfield, New Jersey</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="Peltz, Stuart W" sort="Peltz, Stuart W" uniqKey="Peltz S" first="Stuart W" last="Peltz">Stuart W. Peltz</name><affiliation wicri:level="2"><nlm:aff id="au24"><institution>PTC Therapeutics</institution><addr-line>South Plainfield, New Jersey, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>South Plainfield, New Jersey</wicri:regionArea><placeName><region type="state">New Jersey</region></placeName></affiliation></author><author><name sortKey="Mcdonald, Craig M" sort="Mcdonald, Craig M" uniqKey="Mcdonald C" first="Craig M" last="Mcdonald">Craig M. Mcdonald</name><affiliation wicri:level="1"><nlm:aff id="au21"><institution>UC Davis Children's Hospital, Lawrence J. Ellison Ambulatory Care Center, Physical Medicine & Rehabilitation</institution><addr-line>4860 Y St., Suite 1700, Sacramento, California, 95817, USA</addr-line></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>4860 Y St., Suite 1700, Sacramento, California, 95817</wicri:regionArea><wicri:noRegion>95817</wicri:noRegion></affiliation></author></analytic><series><title level="j">Muscle & Nerve</title><idno type="ISSN">0148-639X</idno><idno type="eISSN">1097-4598</idno><imprint><date when="2014">2014</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><p><italic>Introduction</italic>: Dystrophinopathy is a rare, severe muscle disorder, and nonsense mutations are found in 13% of cases. Ataluren was developed to enable ribosomal readthrough of premature stop codons in nonsense mutation (nm) genetic disorders. <italic>Methods</italic>: Randomized, double-blind, placebo-controlled study; males ≥5 years with nm-dystrophinopathy received study drug orally 3 times daily, ataluren 10, 10, 20 mg/kg (<italic>N</italic> = 57); ataluren 20, 20, 40 mg/kg (<italic>N</italic> = 60); or placebo (<italic>N</italic> = 57) for 48 weeks. The primary endpoint was change in 6-Minute Walk Distance (6MWD) at Week 48. <italic>Results</italic>: Ataluren was generally well tolerated. The primary endpoint favored ataluren 10, 10, 20 mg/kg versus placebo; the week 48 6MWD Δ = 31.3 meters, <italic>post hoc P</italic> = 0.056. Secondary endpoints (timed function tests) showed meaningful differences between ataluren 10, 10, 20 mg/kg, and placebo. <italic>Conclusions</italic>: As the first investigational new drug targeting the underlying cause of nm-dystrophinopathy, ataluren offers promise as a treatment for this orphan genetic disorder with high unmet medical need. <italic>Muscle Nerve</italic><bold>50</bold>: 477–487, 2014</p></div></front><back><div1 type="bibliography"><listBibl><biblStruct><analytic><author><name sortKey="Dent, Km" uniqKey="Dent K">KM Dent</name></author><author><name sortKey="Dunn, Dm" uniqKey="Dunn D">DM Dunn</name></author><author><name sortKey="Von Niederhausern, Ac" uniqKey="Von Niederhausern A">AC von Niederhausern</name></author><author><name sortKey="Aoyagi, At" uniqKey="Aoyagi A">AT Aoyagi</name></author><author><name sortKey="Kerr, L" uniqKey="Kerr L">L Kerr</name></author><author><name sortKey="Bromberg, Mb" uniqKey="Bromberg M">MB 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sortKey="Munnich, A" uniqKey="Munnich A">A Munnich</name></author><author><name sortKey="Cormier Daire, V" uniqKey="Cormier Daire V">V Cormier-Daire</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Brown, Kj" uniqKey="Brown K">KJ Brown</name></author><author><name sortKey="Marathi, R" uniqKey="Marathi R">R Marathi</name></author><author><name sortKey="Fiorillo, Aa" uniqKey="Fiorillo A">AA Fiorillo</name></author><author><name sortKey="Ciccimaro, Ef" uniqKey="Ciccimaro E">EF Ciccimaro</name></author><author><name sortKey="Sharma, S" uniqKey="Sharma S">S Sharma</name></author><author><name sortKey="Rowlands, Ds" uniqKey="Rowlands D">DS Rowlands</name></author></analytic></biblStruct><biblStruct></biblStruct></listBibl></div1></back></TEI></record>Pour manipuler ce document sous Unix (Dilib)
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