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Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA).
Oskoui, Maryam; Day, John W; Deconinck, Nicolas; Mazzone, Elena S; Nascimento, Andres; Saito, Kayoko; Vuillerot, Carole; Baranello, Giovanni; Goemans, Nathalie; Kirschner, Janbernd; Kostera-Pruszczyk, Anna; Servais, Laurent; Papp, Gergely; Gorni, Ksenija; Kletzl, Heidemarie; Martin, Carmen; McIver, Tammy; Scalco, Renata S; Staunton, Hannah; Yeung, Wai Yin; Fontoura, Paulo; Mercuri, Eugenio.
Afiliação
  • Oskoui M; Departments of Pediatrics and Neurology and Neurosurgery, McGill University, Montreal, Canada. maryam.oskoui@mcgill.ca.
  • Day JW; Department of Neurology, Stanford University, Palo Alto, CA, USA.
  • Deconinck N; Neuromuscular Reference Center, UZ Gent, Ghent, Belgium.
  • Mazzone ES; Centre de Référence des Maladies Neuromusculaires et Service de Neurologie Pédiatrique, Queen Fabiola Children's University Hospital, Université Libre de Bruxelles, ULB, Brussels, Belgium.
  • Nascimento A; Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy.
  • Saito K; Neuromuscular Unit, Neuropaediatrics Department, Hospital Sant Joan de Déu, Fundacion Sant Joan de Deu, CIBERER-ISC III, Barcelona, Spain.
  • Vuillerot C; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.
  • Baranello G; Department of Pediatric Physical Medicine and Rehabilitation, Hôpital Mère Enfant, CHU-Lyon, Lyon, France.
  • Goemans N; Neuromyogen Institute, CNRS UMR 5310-INSERM U1217, Université de Lyon, Lyon, France.
  • Kirschner J; The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London and Great Ormond Street Hospital Trust, London, UK.
  • Kostera-Pruszczyk A; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
  • Servais L; Neuromuscular Reference Centre, Department of Paediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium.
  • Papp G; Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, Medical Center-University of Freiburg, Freiburg, Germany.
  • Gorni K; Department of Neurology, Medical University of Warsaw, Warsaw, Poland.
  • Kletzl H; I-Motion-Hôpital Armand Trousseau, Paris, France.
  • Martin C; MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK.
  • McIver T; Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, University Hospital Liège and University of Liège, Liège, Belgium.
  • Scalco RS; Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
  • Staunton H; PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
  • Yeung WY; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland.
  • Fontoura P; Roche Products Ltd, Welwyn Garden City, UK.
  • Mercuri E; Roche Products Ltd, Welwyn Garden City, UK.
J Neurol ; 270(5): 2531-2546, 2023 May.
Article em En | MEDLINE | ID: mdl-36735057
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal / Atrofias Musculares Espinais da Infância Idioma: En Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Canadá
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal / Atrofias Musculares Espinais da Infância Idioma: En Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Canadá