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Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.
Diaz-Manera, Jordi; Kishnani, Priya S; Kushlaf, Hani; Ladha, Shafeeq; Mozaffar, Tahseen; Straub, Volker; Toscano, Antonio; van der Ploeg, Ans T; Berger, Kenneth I; Clemens, Paula R; Chien, Yin-Hsiu; Day, John W; Illarioshkin, Sergey; Roberts, Mark; Attarian, Shahram; Borges, Joao Lindolfo; Bouhour, Francoise; Choi, Young Chul; Erdem-Ozdamar, Sevim; Goker-Alpan, Ozlem; Kostera-Pruszczyk, Anna; Haack, Kristina An; Hug, Christopher; Huynh-Ba, Olivier; Johnson, Judith; Thibault, Nathan; Zhou, Tianyue; Dimachkie, Mazen M; Schoser, Benedikt.
Afiliación
  • Diaz-Manera J; John Walton Muscular Dystrophy Research Centre, Newcastle University Centre for Life, Newcastle upon Tyne, UK; Neuromuscular Diseases Unit, Neurology Department, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain; Centro de Investigación Biomédica en Red en Enfermedades Raras (CIBERER), Spain. E
  • Kishnani PS; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.
  • Kushlaf H; Department of Neurology and Rehabilitation Medicine, and Department of Pathology and Laboratory Medicine, University of Cincinnati, Cincinnati, OH, USA.
  • Ladha S; Gregory W Fulton ALS and Neuromuscular Center, Barrow Neurological Institute, Phoenix, AZ, USA.
  • Mozaffar T; Department of Neurology, University of California, Irvine, Orange, CA, USA.
  • Straub V; John Walton Muscular Dystrophy Research Centre, Newcastle University Centre for Life, Newcastle upon Tyne, UK.
  • Toscano A; Department of Clinical and Experimental Medicine, Reference Centre for Rare Neuromuscular Disorders, University of Messina, Messina, Italy.
  • van der Ploeg AT; Center for Lysosomal and Metabolic Diseases, Erasmus MC, University Medical Center, Rotterdam, Netherlands.
  • Berger KI; Division of Pulmonary, Critical Care and Sleep Medicine, New York University Grossman School of Medicine, New York, NY, USA; André Cournand Pulmonary Physiology Laboratory, Bellevue Hospital, New York, NY, USA.
  • Clemens PR; Department of Neurology, University of Pittsburgh, Pittsburgh, PA, USA; Department of Veterans Affairs Medical Center, Pittsburgh, PA, USA.
  • Chien YH; Department of Medical Genetics and Pediatrics, National Taiwan University Hospital, Taipei, Taiwan.
  • Day JW; Department of Neurology, and Department of Pediatrics, Stanford University, Stanford, CA, USA.
  • Illarioshkin S; Research Center of Neurology, Moscow, Russia.
  • Roberts M; Salford Royal NHS Foundation Trust, Salford, UK.
  • Attarian S; Referral Centre for Neuromuscular Diseases and ALS, Hôpital La Timone, Marseille, France.
  • Borges JL; Clinical Research Centre of Brazil, Brasilia, Brazil.
  • Bouhour F; Referral Centre for Neuromuscular Diseases, Hopîtal Neurologique, Lyon-Bron, France.
  • Choi YC; Gangnam Severance Hospital, Yonsei University, College of Medicine, Seoul, South Korea.
  • Erdem-Ozdamar S; Hacettepe University Department of Neurology, Ankara, Turkey.
  • Goker-Alpan O; Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), Fairfax, VA, USA.
  • Kostera-Pruszczyk A; Department of Neurology, Medical University of Warsaw, Warsaw, Poland, ERN EURO-NMD.
  • Haack KA; Sanofi Genzyme, Shanghai, China.
  • Hug C; Sanofi Genzyme, Cambridge, MA, USA.
  • Huynh-Ba O; Sanofi Genzyme, Chilly-Mazarin, France.
  • Johnson J; Sanofi Genzyme, Cambridge, MA, USA.
  • Thibault N; Sanofi Genzyme, Cambridge, MA, USA.
  • Zhou T; Sanofi Genzyme, Cambridge, MA, USA.
  • Dimachkie MM; University of Kansas Medical Center, Department of Neurology, Kansas City, KS, USA.
  • Schoser B; Friedrich-Baur-Institute, Department of Neurology, LMU Klinikum München, Munich, Germany.
Lancet Neurol ; 20(12): 1012-1026, 2021 12.
Article en En | MEDLINE | ID: mdl-34800399
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedad del Almacenamiento de Glucógeno Tipo II / Alfa-Glucosidasas Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Child, preschool / Humans Idioma: En Revista: Lancet Neurol Asunto de la revista: NEUROLOGIA Año: 2021 Tipo del documento: Article
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedad del Almacenamiento de Glucógeno Tipo II / Alfa-Glucosidasas Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Child, preschool / Humans Idioma: En Revista: Lancet Neurol Asunto de la revista: NEUROLOGIA Año: 2021 Tipo del documento: Article