A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency.
Identifieur interne : 003259 ( Main/Exploration ); précédent : 003258; suivant : 003260A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency.
Auteurs : Barbara K. Burton [République tchèque] ; Manisha Balwani ; François Feillet ; Ivo Bari ; T Andrew Burrow ; Carmen Camarena Grande ; Mahmut Coker ; Alejandra Consuelo-Sánchez ; Patrick Deegan ; Maja Di Rocco ; Gregory M. Enns ; Richard Erbe ; Fatih Ezgu ; Can Ficicioglu ; Katryn N. Furuya ; John Kane ; Christina Laukaitis ; Eugen Mengel ; Edward G. Neilan ; Scott Nightingale ; Heidi Peters ; Maurizio Scarpa ; K Otfried Schwab ; Vratislav Smolka ; Vassili Valayannopoulos ; Marnie Wood ; Zachary Goodman ; Yijun Yang ; Stephen Eckert ; Sandra Rojas-Caro ; Anthony G. QuinnSource :
- The New England journal of medicine [ 1533-4406 ] ; 2015.
Descripteurs français
- KwdFr :
- Adolescent, Adulte, Adulte d'âge moyen, Alanine transaminase (sang), Biopsie, Cholestérol HDL (sang), Cholestérol LDL (sang), Dyslipidémies (génétique), Dyslipidémies (traitement médicamenteux), Enfant, Enfant d'âge préscolaire, Femelle, Foie (), Foie (anatomopathologie), Humains, Jeune adulte, Maladie de Wolman (sang), Maladie de Wolman (traitement médicamenteux), Mâle, Méthode en double aveugle, Sterol Esterase (effets indésirables), Sterol Esterase (pharmacologie), Sterol Esterase (usage thérapeutique), Sujet âgé.
- MESH :
- anatomopathologie : Foie.
- effets indésirables : Sterol Esterase.
- génétique : Dyslipidémies.
- pharmacologie : Sterol Esterase.
- sang : Alanine transaminase, Cholestérol HDL, Cholestérol LDL, Maladie de Wolman.
- traitement médicamenteux : Dyslipidémies, Maladie de Wolman.
- usage thérapeutique : Sterol Esterase.
- Adolescent, Adulte, Adulte d'âge moyen, Biopsie, Enfant, Enfant d'âge préscolaire, Femelle, Foie, Humains, Jeune adulte, Mâle, Méthode en double aveugle, Sujet âgé.
English descriptors
- KwdEn :
- Adolescent, Adult, Aged, Alanine Transaminase (blood), Biopsy, Child, Child, Preschool, Cholesterol, HDL (blood), Cholesterol, LDL (blood), Double-Blind Method, Dyslipidemias (drug therapy), Dyslipidemias (genetics), Female, Humans, Liver (drug effects), Liver (pathology), Male, Middle Aged, Sterol Esterase (adverse effects), Sterol Esterase (pharmacology), Sterol Esterase (therapeutic use), Wolman Disease (blood), Wolman Disease (drug therapy), Young Adult.
- MESH :
- chemical , adverse effects : Sterol Esterase.
- chemical , blood : Alanine Transaminase, Cholesterol, HDL, Cholesterol, LDL.
- blood : Wolman Disease.
- drug effects : Liver.
- drug therapy : Dyslipidemias, Wolman Disease.
- genetics : Dyslipidemias.
- pathology : Liver.
- chemical , pharmacology : Sterol Esterase.
- chemical , therapeutic use : Sterol Esterase.
- Adolescent, Adult, Aged, Biopsy, Child, Child, Preschool, Double-Blind Method, Female, Humans, Male, Middle Aged, Young Adult.
Abstract
Lysosomal acid lipase is an essential lipid-metabolizing enzyme that breaks down endocytosed lipid particles and regulates lipid metabolism. We conducted a phase 3 trial of enzyme-replacement therapy in children and adults with lysosomal acid lipase deficiency, an underappreciated cause of cirrhosis and severe dyslipidemia.
DOI: 10.1056/NEJMoa1501365
PubMed: 26352813
Affiliations:
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Le document en format XML
<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency.</title><author><name sortKey="Burton, Barbara K" sort="Burton, Barbara K" uniqKey="Burton B" first="Barbara K" last="Burton">Barbara K. Burton</name><affiliation wicri:level="1"><nlm:affiliation>From the Northwestern University Feinberg School of Medicine and the Ann and Robert H. Lurie Children's Hospital, Chicago (B.K.B.); Icahn School of Medicine, Mount Sinai, New York (M.B.), and Women and Children's Hospital of Buffalo, Buffalo (R.E.) - both in New York; Centre Hospitalier Universitaire Brabois-Hôpital d'Enfants, Vandoeuvre-lès-Nancy (F.F.), and University Hospital Necker-Enfants Malades and Imagine Institute, Paris (V.V.) - both in France; University Hospital Center Zagreb and University of Zagreb, School of Medicine, Zagreb, Croatia (I.B.); Cincinnati Children's Hospital Medical Center, Cincinnati (T.A.B.); Hospital Universitario La Paz, Madrid (C.C.G.); Ege University Medical Faculty, Izmir (M.C.), and Gazi University Medical Faculty, Ankara (F.E.) - both in Turkey; Hospital Infantil de México Federico Gómez, Mexico City (A.C.-S.); Cambridge University Hospitals, Cambridge, United Kingdom (P.D.); Unit of Rare Diseases, Department of Pediatrics, Gaslini Institute, Genoa (M.D.R.), and University of Padua, Padua (M.S.) - both in Italy; Stanford University, Palo Alto (G.M.E.), and University of California, San Francisco, San Francisco ( J.K.) - both in California; Children's Hospital of Philadelphia, Philadelphia (C.F.); Alfred I. duPont Hospital for Children, Wilmington, DE (K.N.F.); University of Arizona Cancer Center, Tucson (C.L.); Villa Metabolica, Center of Pediatric and Adolescent Medicine, University of Mainz, Mainz (E.M.), and University Hospital Freiburg, Freiburg (K.O.S.) - both in Germany; Boston Children's Hospital, Boston (E.G.N.), and Synageva BioPharma, Lexington (Y.Y., S.E., S.R.-C., A.G.Q.) - both in Massachusetts; John Hunter Children's Hospital, and Discipline of Paediatrics and Child Health, University of Newcastle, Newcastle, NSW (S.N.), Royal Children's Hospital, Parkville, VIC (H.P.), and Royal Brisbane and Women's Hospital, Brisbane, QLD (M.W.) - all in Australia; Faculty Hospital, Palacky University, Olomouc, Czech Republic</nlm:affiliation><country xml:lang="fr">République tchèque</country><wicri:regionArea>From the Northwestern University Feinberg School of Medicine and the Ann and Robert H. Lurie Children's Hospital, Chicago (B.K.B.); Icahn School of Medicine, Mount Sinai, New York (M.B.), and Women and Children's Hospital of Buffalo, Buffalo (R.E.) - both in New York; Centre Hospitalier Universitaire Brabois-Hôpital d'Enfants, Vandoeuvre-lès-Nancy (F.F.), and University Hospital Necker-Enfants Malades and Imagine Institute, Paris (V.V.) - both in France; University Hospital Center Zagreb and University of Zagreb, School of Medicine, Zagreb, Croatia (I.B.); Cincinnati Children's Hospital Medical Center, Cincinnati (T.A.B.); Hospital Universitario La Paz, Madrid (C.C.G.); Ege University Medical Faculty, Izmir (M.C.), and Gazi University Medical Faculty, Ankara (F.E.) - both in Turkey; Hospital Infantil de México Federico Gómez, Mexico City (A.C.-S.); Cambridge University Hospitals, Cambridge, United Kingdom (P.D.); Unit of Rare Diseases, Department of Pediatrics, Gaslini Institute, Genoa (M.D.R.), and University of Padua, Padua (M.S.) - both in Italy; Stanford University, Palo Alto (G.M.E.), and University of California, San Francisco, San Francisco ( J.K.) - both in California; Children's Hospital of Philadelphia, Philadelphia (C.F.); Alfred I. duPont Hospital for Children, Wilmington, DE (K.N.F.); University of Arizona Cancer Center, Tucson (C.L.); Villa Metabolica, Center of Pediatric and Adolescent Medicine, University of Mainz, Mainz (E.M.), and University Hospital Freiburg, Freiburg (K.O.S.) - both in Germany; Boston Children's Hospital, Boston (E.G.N.), and Synageva BioPharma, Lexington (Y.Y., S.E., S.R.-C., A.G.Q.) - both in Massachusetts; John Hunter Children's Hospital, and Discipline of Paediatrics and Child Health, University of Newcastle, Newcastle, NSW (S.N.), Royal Children's Hospital, Parkville, VIC (H.P.), and Royal Brisbane and Women's Hospital, Brisbane, QLD (M.W.) - all in Australia; Faculty Hospital, Palacky University, Olomouc</wicri:regionArea><wicri:noRegion>Olomouc</wicri:noRegion></affiliation></author><author><name sortKey="Balwani, Manisha" sort="Balwani, Manisha" uniqKey="Balwani M" first="Manisha" last="Balwani">Manisha Balwani</name></author><author><name sortKey="Feillet, Francois" sort="Feillet, Francois" uniqKey="Feillet F" first="François" last="Feillet">François Feillet</name></author><author><name sortKey="Bari, Ivo" sort="Bari, Ivo" uniqKey="Bari I" first="Ivo" last="Bari">Ivo Bari</name></author><author><name sortKey="Burrow, T Andrew" sort="Burrow, T Andrew" uniqKey="Burrow T" first="T Andrew" last="Burrow">T Andrew Burrow</name></author><author><name sortKey="Camarena Grande, Carmen" sort="Camarena Grande, Carmen" uniqKey="Camarena Grande C" first="Carmen" last="Camarena Grande">Carmen Camarena Grande</name></author><author><name sortKey="Coker, Mahmut" sort="Coker, Mahmut" uniqKey="Coker M" first="Mahmut" last="Coker">Mahmut Coker</name></author><author><name sortKey="Consuelo Sanchez, Alejandra" sort="Consuelo Sanchez, Alejandra" uniqKey="Consuelo Sanchez A" first="Alejandra" last="Consuelo-Sánchez">Alejandra Consuelo-Sánchez</name></author><author><name sortKey="Deegan, Patrick" sort="Deegan, Patrick" uniqKey="Deegan P" first="Patrick" last="Deegan">Patrick Deegan</name></author><author><name sortKey="Di Rocco, Maja" sort="Di Rocco, Maja" uniqKey="Di Rocco M" first="Maja" last="Di Rocco">Maja Di Rocco</name></author><author><name sortKey="Enns, Gregory M" sort="Enns, Gregory M" uniqKey="Enns G" first="Gregory M" last="Enns">Gregory M. Enns</name></author><author><name sortKey="Erbe, Richard" sort="Erbe, Richard" uniqKey="Erbe R" first="Richard" last="Erbe">Richard Erbe</name></author><author><name sortKey="Ezgu, Fatih" sort="Ezgu, Fatih" uniqKey="Ezgu F" first="Fatih" last="Ezgu">Fatih Ezgu</name></author><author><name sortKey="Ficicioglu, Can" sort="Ficicioglu, Can" uniqKey="Ficicioglu C" first="Can" last="Ficicioglu">Can Ficicioglu</name></author><author><name sortKey="Furuya, Katryn N" sort="Furuya, Katryn N" uniqKey="Furuya K" first="Katryn N" last="Furuya">Katryn N. Furuya</name></author><author><name sortKey="Kane, John" sort="Kane, John" uniqKey="Kane J" first="John" last="Kane">John Kane</name></author><author><name sortKey="Laukaitis, Christina" sort="Laukaitis, Christina" uniqKey="Laukaitis C" first="Christina" last="Laukaitis">Christina Laukaitis</name></author><author><name sortKey="Mengel, Eugen" sort="Mengel, Eugen" uniqKey="Mengel E" first="Eugen" last="Mengel">Eugen Mengel</name></author><author><name sortKey="Neilan, Edward G" sort="Neilan, Edward G" uniqKey="Neilan E" first="Edward G" last="Neilan">Edward G. Neilan</name></author><author><name sortKey="Nightingale, Scott" sort="Nightingale, Scott" uniqKey="Nightingale S" first="Scott" last="Nightingale">Scott Nightingale</name></author><author><name sortKey="Peters, Heidi" sort="Peters, Heidi" uniqKey="Peters H" first="Heidi" last="Peters">Heidi Peters</name></author><author><name sortKey="Scarpa, Maurizio" sort="Scarpa, Maurizio" uniqKey="Scarpa M" first="Maurizio" last="Scarpa">Maurizio Scarpa</name></author><author><name sortKey="Schwab, K Otfried" sort="Schwab, K Otfried" uniqKey="Schwab K" first="K Otfried" last="Schwab">K Otfried Schwab</name></author><author><name sortKey="Smolka, Vratislav" sort="Smolka, Vratislav" uniqKey="Smolka V" first="Vratislav" last="Smolka">Vratislav Smolka</name></author><author><name sortKey="Valayannopoulos, Vassili" sort="Valayannopoulos, Vassili" uniqKey="Valayannopoulos V" first="Vassili" last="Valayannopoulos">Vassili Valayannopoulos</name></author><author><name sortKey="Wood, Marnie" sort="Wood, Marnie" uniqKey="Wood M" first="Marnie" last="Wood">Marnie Wood</name></author><author><name sortKey="Goodman, Zachary" sort="Goodman, Zachary" uniqKey="Goodman Z" first="Zachary" last="Goodman">Zachary Goodman</name></author><author><name sortKey="Yang, Yijun" sort="Yang, Yijun" uniqKey="Yang Y" first="Yijun" last="Yang">Yijun Yang</name></author><author><name sortKey="Eckert, Stephen" sort="Eckert, Stephen" uniqKey="Eckert S" first="Stephen" last="Eckert">Stephen Eckert</name></author><author><name sortKey="Rojas Caro, Sandra" sort="Rojas Caro, Sandra" uniqKey="Rojas Caro S" first="Sandra" last="Rojas-Caro">Sandra Rojas-Caro</name></author><author><name sortKey="Quinn, Anthony G" sort="Quinn, Anthony G" uniqKey="Quinn A" first="Anthony G" last="Quinn">Anthony G. Quinn</name></author></titleStmt><publicationStmt><idno type="wicri:source">PubMed</idno><date when="2015">2015</date><idno type="RBID">pubmed:26352813</idno><idno type="pmid">26352813</idno><idno type="doi">10.1056/NEJMoa1501365</idno><idno type="wicri:Area/PubMed/Corpus">002996</idno><idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Corpus" wicri:corpus="PubMed">002996</idno><idno type="wicri:Area/PubMed/Curation">002926</idno><idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Curation">002926</idno><idno type="wicri:Area/PubMed/Checkpoint">002926</idno><idno type="wicri:explorRef" wicri:stream="Checkpoint" wicri:step="PubMed">002926</idno><idno type="wicri:Area/Ncbi/Merge">002A39</idno><idno type="wicri:Area/Ncbi/Curation">002A39</idno><idno type="wicri:Area/Ncbi/Checkpoint">002A39</idno><idno type="wicri:Area/Main/Merge">003260</idno><idno type="wicri:Area/Main/Curation">003259</idno><idno type="wicri:Area/Main/Exploration">003259</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en">A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency.</title><author><name sortKey="Burton, Barbara K" sort="Burton, Barbara K" uniqKey="Burton B" first="Barbara K" last="Burton">Barbara K. Burton</name><affiliation wicri:level="1"><nlm:affiliation>From the Northwestern University Feinberg School of Medicine and the Ann and Robert H. Lurie Children's Hospital, Chicago (B.K.B.); Icahn School of Medicine, Mount Sinai, New York (M.B.), and Women and Children's Hospital of Buffalo, Buffalo (R.E.) - both in New York; Centre Hospitalier Universitaire Brabois-Hôpital d'Enfants, Vandoeuvre-lès-Nancy (F.F.), and University Hospital Necker-Enfants Malades and Imagine Institute, Paris (V.V.) - both in France; University Hospital Center Zagreb and University of Zagreb, School of Medicine, Zagreb, Croatia (I.B.); Cincinnati Children's Hospital Medical Center, Cincinnati (T.A.B.); Hospital Universitario La Paz, Madrid (C.C.G.); Ege University Medical Faculty, Izmir (M.C.), and Gazi University Medical Faculty, Ankara (F.E.) - both in Turkey; Hospital Infantil de México Federico Gómez, Mexico City (A.C.-S.); Cambridge University Hospitals, Cambridge, United Kingdom (P.D.); Unit of Rare Diseases, Department of Pediatrics, Gaslini Institute, Genoa (M.D.R.), and University of Padua, Padua (M.S.) - both in Italy; Stanford University, Palo Alto (G.M.E.), and University of California, San Francisco, San Francisco ( J.K.) - both in California; Children's Hospital of Philadelphia, Philadelphia (C.F.); Alfred I. duPont Hospital for Children, Wilmington, DE (K.N.F.); University of Arizona Cancer Center, Tucson (C.L.); Villa Metabolica, Center of Pediatric and Adolescent Medicine, University of Mainz, Mainz (E.M.), and University Hospital Freiburg, Freiburg (K.O.S.) - both in Germany; Boston Children's Hospital, Boston (E.G.N.), and Synageva BioPharma, Lexington (Y.Y., S.E., S.R.-C., A.G.Q.) - both in Massachusetts; John Hunter Children's Hospital, and Discipline of Paediatrics and Child Health, University of Newcastle, Newcastle, NSW (S.N.), Royal Children's Hospital, Parkville, VIC (H.P.), and Royal Brisbane and Women's Hospital, Brisbane, QLD (M.W.) - all in Australia; Faculty Hospital, Palacky University, Olomouc, Czech Republic</nlm:affiliation><country xml:lang="fr">République tchèque</country><wicri:regionArea>From the Northwestern University Feinberg School of Medicine and the Ann and Robert H. Lurie Children's Hospital, Chicago (B.K.B.); Icahn School of Medicine, Mount Sinai, New York (M.B.), and Women and Children's Hospital of Buffalo, Buffalo (R.E.) - both in New York; Centre Hospitalier Universitaire Brabois-Hôpital d'Enfants, Vandoeuvre-lès-Nancy (F.F.), and University Hospital Necker-Enfants Malades and Imagine Institute, Paris (V.V.) - both in France; University Hospital Center Zagreb and University of Zagreb, School of Medicine, Zagreb, Croatia (I.B.); Cincinnati Children's Hospital Medical Center, Cincinnati (T.A.B.); Hospital Universitario La Paz, Madrid (C.C.G.); Ege University Medical Faculty, Izmir (M.C.), and Gazi University Medical Faculty, Ankara (F.E.) - both in Turkey; Hospital Infantil de México Federico Gómez, Mexico City (A.C.-S.); Cambridge University Hospitals, Cambridge, United Kingdom (P.D.); Unit of Rare Diseases, Department of Pediatrics, Gaslini Institute, Genoa (M.D.R.), and University of Padua, Padua (M.S.) - both in Italy; Stanford University, Palo Alto (G.M.E.), and University of California, San Francisco, San Francisco ( J.K.) - both in California; Children's Hospital of Philadelphia, Philadelphia (C.F.); Alfred I. duPont Hospital for Children, Wilmington, DE (K.N.F.); University of Arizona Cancer Center, Tucson (C.L.); Villa Metabolica, Center of Pediatric and Adolescent Medicine, University of Mainz, Mainz (E.M.), and University Hospital Freiburg, Freiburg (K.O.S.) - both in Germany; Boston Children's Hospital, Boston (E.G.N.), and Synageva BioPharma, Lexington (Y.Y., S.E., S.R.-C., A.G.Q.) - both in Massachusetts; John Hunter Children's Hospital, and Discipline of Paediatrics and Child Health, University of Newcastle, Newcastle, NSW (S.N.), Royal Children's Hospital, Parkville, VIC (H.P.), and Royal Brisbane and Women's Hospital, Brisbane, QLD (M.W.) - all in Australia; Faculty Hospital, Palacky University, Olomouc</wicri:regionArea><wicri:noRegion>Olomouc</wicri:noRegion></affiliation></author><author><name sortKey="Balwani, Manisha" sort="Balwani, Manisha" uniqKey="Balwani M" first="Manisha" last="Balwani">Manisha Balwani</name></author><author><name sortKey="Feillet, Francois" sort="Feillet, Francois" uniqKey="Feillet F" first="François" last="Feillet">François Feillet</name></author><author><name sortKey="Bari, Ivo" sort="Bari, Ivo" uniqKey="Bari I" first="Ivo" last="Bari">Ivo Bari</name></author><author><name sortKey="Burrow, T Andrew" sort="Burrow, T Andrew" uniqKey="Burrow T" first="T Andrew" last="Burrow">T Andrew Burrow</name></author><author><name sortKey="Camarena Grande, Carmen" sort="Camarena Grande, Carmen" uniqKey="Camarena Grande C" first="Carmen" last="Camarena Grande">Carmen Camarena Grande</name></author><author><name sortKey="Coker, Mahmut" sort="Coker, Mahmut" uniqKey="Coker M" first="Mahmut" last="Coker">Mahmut Coker</name></author><author><name sortKey="Consuelo Sanchez, Alejandra" sort="Consuelo Sanchez, Alejandra" uniqKey="Consuelo Sanchez A" first="Alejandra" last="Consuelo-Sánchez">Alejandra Consuelo-Sánchez</name></author><author><name sortKey="Deegan, Patrick" sort="Deegan, Patrick" uniqKey="Deegan P" first="Patrick" last="Deegan">Patrick Deegan</name></author><author><name sortKey="Di Rocco, Maja" sort="Di Rocco, Maja" uniqKey="Di Rocco M" first="Maja" last="Di Rocco">Maja Di Rocco</name></author><author><name sortKey="Enns, Gregory M" sort="Enns, Gregory M" uniqKey="Enns G" first="Gregory M" last="Enns">Gregory M. Enns</name></author><author><name sortKey="Erbe, Richard" sort="Erbe, Richard" uniqKey="Erbe R" first="Richard" last="Erbe">Richard Erbe</name></author><author><name sortKey="Ezgu, Fatih" sort="Ezgu, Fatih" uniqKey="Ezgu F" first="Fatih" last="Ezgu">Fatih Ezgu</name></author><author><name sortKey="Ficicioglu, Can" sort="Ficicioglu, Can" uniqKey="Ficicioglu C" first="Can" last="Ficicioglu">Can Ficicioglu</name></author><author><name sortKey="Furuya, Katryn N" sort="Furuya, Katryn N" uniqKey="Furuya K" first="Katryn N" last="Furuya">Katryn N. Furuya</name></author><author><name sortKey="Kane, John" sort="Kane, John" uniqKey="Kane J" first="John" last="Kane">John Kane</name></author><author><name sortKey="Laukaitis, Christina" sort="Laukaitis, Christina" uniqKey="Laukaitis C" first="Christina" last="Laukaitis">Christina Laukaitis</name></author><author><name sortKey="Mengel, Eugen" sort="Mengel, Eugen" uniqKey="Mengel E" first="Eugen" last="Mengel">Eugen Mengel</name></author><author><name sortKey="Neilan, Edward G" sort="Neilan, Edward G" uniqKey="Neilan E" first="Edward G" last="Neilan">Edward G. Neilan</name></author><author><name sortKey="Nightingale, Scott" sort="Nightingale, Scott" uniqKey="Nightingale S" first="Scott" last="Nightingale">Scott Nightingale</name></author><author><name sortKey="Peters, Heidi" sort="Peters, Heidi" uniqKey="Peters H" first="Heidi" last="Peters">Heidi Peters</name></author><author><name sortKey="Scarpa, Maurizio" sort="Scarpa, Maurizio" uniqKey="Scarpa M" first="Maurizio" last="Scarpa">Maurizio Scarpa</name></author><author><name sortKey="Schwab, K Otfried" sort="Schwab, K Otfried" uniqKey="Schwab K" first="K Otfried" last="Schwab">K Otfried Schwab</name></author><author><name sortKey="Smolka, Vratislav" sort="Smolka, Vratislav" uniqKey="Smolka V" first="Vratislav" last="Smolka">Vratislav Smolka</name></author><author><name sortKey="Valayannopoulos, Vassili" sort="Valayannopoulos, Vassili" uniqKey="Valayannopoulos V" first="Vassili" last="Valayannopoulos">Vassili Valayannopoulos</name></author><author><name sortKey="Wood, Marnie" sort="Wood, Marnie" uniqKey="Wood M" first="Marnie" last="Wood">Marnie Wood</name></author><author><name sortKey="Goodman, Zachary" sort="Goodman, Zachary" uniqKey="Goodman Z" first="Zachary" last="Goodman">Zachary Goodman</name></author><author><name sortKey="Yang, Yijun" sort="Yang, Yijun" uniqKey="Yang Y" first="Yijun" last="Yang">Yijun Yang</name></author><author><name sortKey="Eckert, Stephen" sort="Eckert, Stephen" uniqKey="Eckert S" first="Stephen" last="Eckert">Stephen Eckert</name></author><author><name sortKey="Rojas Caro, Sandra" sort="Rojas Caro, Sandra" uniqKey="Rojas Caro S" first="Sandra" last="Rojas-Caro">Sandra Rojas-Caro</name></author><author><name sortKey="Quinn, Anthony G" sort="Quinn, Anthony G" uniqKey="Quinn A" first="Anthony G" last="Quinn">Anthony G. Quinn</name></author></analytic><series><title level="j">The New England journal of medicine</title><idno type="eISSN">1533-4406</idno><imprint><date when="2015" type="published">2015</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Adolescent</term><term>Adult</term><term>Aged</term><term>Alanine Transaminase (blood)</term><term>Biopsy</term><term>Child</term><term>Child, Preschool</term><term>Cholesterol, HDL (blood)</term><term>Cholesterol, LDL (blood)</term><term>Double-Blind Method</term><term>Dyslipidemias (drug therapy)</term><term>Dyslipidemias (genetics)</term><term>Female</term><term>Humans</term><term>Liver (drug effects)</term><term>Liver (pathology)</term><term>Male</term><term>Middle Aged</term><term>Sterol Esterase (adverse effects)</term><term>Sterol Esterase (pharmacology)</term><term>Sterol Esterase (therapeutic use)</term><term>Wolman Disease (blood)</term><term>Wolman Disease (drug therapy)</term><term>Young Adult</term></keywords><keywords scheme="KwdFr" xml:lang="fr"><term>Adolescent</term><term>Adulte</term><term>Adulte d'âge moyen</term><term>Alanine transaminase (sang)</term><term>Biopsie</term><term>Cholestérol HDL (sang)</term><term>Cholestérol LDL (sang)</term><term>Dyslipidémies (génétique)</term><term>Dyslipidémies (traitement médicamenteux)</term><term>Enfant</term><term>Enfant d'âge préscolaire</term><term>Femelle</term><term>Foie ()</term><term>Foie (anatomopathologie)</term><term>Humains</term><term>Jeune adulte</term><term>Maladie de Wolman (sang)</term><term>Maladie de Wolman (traitement médicamenteux)</term><term>Mâle</term><term>Méthode en double aveugle</term><term>Sterol Esterase (effets indésirables)</term><term>Sterol Esterase (pharmacologie)</term><term>Sterol Esterase (usage thérapeutique)</term><term>Sujet âgé</term></keywords><keywords scheme="MESH" type="chemical" qualifier="adverse effects" xml:lang="en"><term>Sterol Esterase</term></keywords><keywords scheme="MESH" type="chemical" qualifier="blood" xml:lang="en"><term>Alanine Transaminase</term><term>Cholesterol, HDL</term><term>Cholesterol, LDL</term></keywords><keywords scheme="MESH" qualifier="anatomopathologie" xml:lang="fr"><term>Foie</term></keywords><keywords scheme="MESH" qualifier="blood" xml:lang="en"><term>Wolman Disease</term></keywords><keywords scheme="MESH" qualifier="drug effects" xml:lang="en"><term>Liver</term></keywords><keywords scheme="MESH" qualifier="drug therapy" xml:lang="en"><term>Dyslipidemias</term><term>Wolman Disease</term></keywords><keywords scheme="MESH" qualifier="effets indésirables" xml:lang="fr"><term>Sterol Esterase</term></keywords><keywords scheme="MESH" qualifier="genetics" xml:lang="en"><term>Dyslipidemias</term></keywords><keywords scheme="MESH" qualifier="génétique" xml:lang="fr"><term>Dyslipidémies</term></keywords><keywords scheme="MESH" qualifier="pathology" xml:lang="en"><term>Liver</term></keywords><keywords scheme="MESH" qualifier="pharmacologie" xml:lang="fr"><term>Sterol Esterase</term></keywords><keywords scheme="MESH" type="chemical" qualifier="pharmacology" xml:lang="en"><term>Sterol Esterase</term></keywords><keywords scheme="MESH" qualifier="sang" xml:lang="fr"><term>Alanine transaminase</term><term>Cholestérol HDL</term><term>Cholestérol LDL</term><term>Maladie de Wolman</term></keywords><keywords scheme="MESH" type="chemical" qualifier="therapeutic use" xml:lang="en"><term>Sterol Esterase</term></keywords><keywords scheme="MESH" qualifier="traitement médicamenteux" xml:lang="fr"><term>Dyslipidémies</term><term>Maladie de Wolman</term></keywords><keywords scheme="MESH" qualifier="usage thérapeutique" xml:lang="fr"><term>Sterol Esterase</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Adolescent</term><term>Adult</term><term>Aged</term><term>Biopsy</term><term>Child</term><term>Child, Preschool</term><term>Double-Blind Method</term><term>Female</term><term>Humans</term><term>Male</term><term>Middle Aged</term><term>Young Adult</term></keywords><keywords scheme="MESH" xml:lang="fr"><term>Adolescent</term><term>Adulte</term><term>Adulte d'âge moyen</term><term>Biopsie</term><term>Enfant</term><term>Enfant d'âge préscolaire</term><term>Femelle</term><term>Foie</term><term>Humains</term><term>Jeune adulte</term><term>Mâle</term><term>Méthode en double aveugle</term><term>Sujet âgé</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Lysosomal acid lipase is an essential lipid-metabolizing enzyme that breaks down endocytosed lipid particles and regulates lipid metabolism. We conducted a phase 3 trial of enzyme-replacement therapy in children and adults with lysosomal acid lipase deficiency, an underappreciated cause of cirrhosis and severe dyslipidemia.</div></front></TEI><affiliations><list><country><li>République tchèque</li></country></list><tree><noCountry><name sortKey="Balwani, Manisha" sort="Balwani, Manisha" uniqKey="Balwani M" first="Manisha" last="Balwani">Manisha Balwani</name><name sortKey="Bari, Ivo" sort="Bari, Ivo" uniqKey="Bari I" first="Ivo" last="Bari">Ivo Bari</name><name sortKey="Burrow, T Andrew" sort="Burrow, T Andrew" uniqKey="Burrow T" first="T Andrew" last="Burrow">T Andrew Burrow</name><name sortKey="Camarena Grande, Carmen" sort="Camarena Grande, Carmen" uniqKey="Camarena Grande C" first="Carmen" last="Camarena Grande">Carmen Camarena Grande</name><name sortKey="Coker, Mahmut" sort="Coker, Mahmut" uniqKey="Coker M" first="Mahmut" last="Coker">Mahmut Coker</name><name sortKey="Consuelo Sanchez, Alejandra" sort="Consuelo Sanchez, Alejandra" uniqKey="Consuelo Sanchez A" first="Alejandra" last="Consuelo-Sánchez">Alejandra Consuelo-Sánchez</name><name sortKey="Deegan, Patrick" sort="Deegan, Patrick" uniqKey="Deegan P" first="Patrick" last="Deegan">Patrick Deegan</name><name sortKey="Di Rocco, Maja" sort="Di Rocco, Maja" uniqKey="Di Rocco M" first="Maja" last="Di Rocco">Maja Di Rocco</name><name sortKey="Eckert, Stephen" sort="Eckert, Stephen" uniqKey="Eckert S" first="Stephen" last="Eckert">Stephen Eckert</name><name sortKey="Enns, Gregory M" sort="Enns, Gregory M" uniqKey="Enns G" first="Gregory M" last="Enns">Gregory M. Enns</name><name sortKey="Erbe, Richard" sort="Erbe, Richard" uniqKey="Erbe R" first="Richard" last="Erbe">Richard Erbe</name><name sortKey="Ezgu, Fatih" sort="Ezgu, Fatih" uniqKey="Ezgu F" first="Fatih" last="Ezgu">Fatih Ezgu</name><name sortKey="Feillet, Francois" sort="Feillet, Francois" uniqKey="Feillet F" first="François" last="Feillet">François Feillet</name><name sortKey="Ficicioglu, Can" sort="Ficicioglu, Can" uniqKey="Ficicioglu C" first="Can" last="Ficicioglu">Can Ficicioglu</name><name sortKey="Furuya, Katryn N" sort="Furuya, Katryn N" uniqKey="Furuya K" first="Katryn N" last="Furuya">Katryn N. Furuya</name><name sortKey="Goodman, Zachary" sort="Goodman, Zachary" uniqKey="Goodman Z" first="Zachary" last="Goodman">Zachary Goodman</name><name sortKey="Kane, John" sort="Kane, John" uniqKey="Kane J" first="John" last="Kane">John Kane</name><name sortKey="Laukaitis, Christina" sort="Laukaitis, Christina" uniqKey="Laukaitis C" first="Christina" last="Laukaitis">Christina Laukaitis</name><name sortKey="Mengel, Eugen" sort="Mengel, Eugen" uniqKey="Mengel E" first="Eugen" last="Mengel">Eugen Mengel</name><name sortKey="Neilan, Edward G" sort="Neilan, Edward G" uniqKey="Neilan E" first="Edward G" last="Neilan">Edward G. Neilan</name><name sortKey="Nightingale, Scott" sort="Nightingale, Scott" uniqKey="Nightingale S" first="Scott" last="Nightingale">Scott Nightingale</name><name sortKey="Peters, Heidi" sort="Peters, Heidi" uniqKey="Peters H" first="Heidi" last="Peters">Heidi Peters</name><name sortKey="Quinn, Anthony G" sort="Quinn, Anthony G" uniqKey="Quinn A" first="Anthony G" last="Quinn">Anthony G. Quinn</name><name sortKey="Rojas Caro, Sandra" sort="Rojas Caro, Sandra" uniqKey="Rojas Caro S" first="Sandra" last="Rojas-Caro">Sandra Rojas-Caro</name><name sortKey="Scarpa, Maurizio" sort="Scarpa, Maurizio" uniqKey="Scarpa M" first="Maurizio" last="Scarpa">Maurizio Scarpa</name><name sortKey="Schwab, K Otfried" sort="Schwab, K Otfried" uniqKey="Schwab K" first="K Otfried" last="Schwab">K Otfried Schwab</name><name sortKey="Smolka, Vratislav" sort="Smolka, Vratislav" uniqKey="Smolka V" first="Vratislav" last="Smolka">Vratislav Smolka</name><name sortKey="Valayannopoulos, Vassili" sort="Valayannopoulos, Vassili" uniqKey="Valayannopoulos V" first="Vassili" last="Valayannopoulos">Vassili Valayannopoulos</name><name sortKey="Wood, Marnie" sort="Wood, Marnie" uniqKey="Wood M" first="Marnie" last="Wood">Marnie Wood</name><name sortKey="Yang, Yijun" sort="Yang, Yijun" uniqKey="Yang Y" first="Yijun" last="Yang">Yijun Yang</name></noCountry><country name="République tchèque"><noRegion><name sortKey="Burton, Barbara K" sort="Burton, Barbara K" uniqKey="Burton B" first="Barbara K" last="Burton">Barbara K. Burton</name></noRegion></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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