Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry.

Autor: Servais L; MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK.; Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liège, Liège, Belgium., Day JW; Department of Neurology, Stanford University Medical Center, Stanford, CA, USA., De Vivo DC; Departments of Neurology and Pediatrics, Columbia University Irving Medical Center, New York, NY, USA., Kirschner J; Department for Neuropediatrics and Muscle Disease, Medical Center - University of Freiburg, Faculty of Medicine, Freiburg, Germany., Mercuri E; Department of Paediatric Neurology and Nemo Clinical Centre, Catholic University, Rome, Italy., Muntoni F; The Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health & Great Ormond Street Hospital, London, UK.; National Institute of Health Research, Great Ormond Street Hospital Biomedical Research Centre, London, UK., Proud CM; Children's Hospital of The King's Daughters, Norfolk, VA, USA., Shieh PB; Department of Neurology, David Geffen School of Medicine at UCLA, University of California, Los Angeles, Los Angeles, CA, USA., Tizzano EF; Department of Clinical and Molecular Genetics, Hospital Vall d'Hebron, Barcelona, Spain., Quijano-Roy S; Garches Neuromuscular Reference Center, Child Neurology and ICU Department, APHP Raymond Poincare University Hospital (UVSQ Paris Saclay), Garches, France., Desguerre I; Hôpital Necker Enfants Malades, APHP, Paris, France., Saito K; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan., Faulkner E; Novartis Gene Therapies, Inc., Bannockburn, IL, USA.; Institute for Precision and Individualized Therapy, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, IL, USA.; Genomics, Biotech and Emerging Medical Technology Institute, National Association of Managed Care Physicians, Richmond, VA, USA., Benguerba KM; Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland., Raju D; Novartis Gene Therapies, Inc., Bannockburn, IL, USA., LaMarca N; Novartis Gene Therapies, Inc., Bannockburn, IL, USA., Sun R; Novartis Gene Therapies, Inc., Bannockburn, IL, USA., Anderson FA; Center for Outcomes Research, University of Massachusetts Medical School, Worcester, MA, USA., Finkel RS; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA.

Publikováno v: Journal of neuromuscular diseases [J Neuromuscul Dis] 2024; Vol. 11 (2), pp. 425-442.