Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy
This study provides Class IV evidence that intravenous onasemnogene abeparvovec is safe in pediatric patients with SMA who weigh 8.5-21 kg.
Authors
- Yin‐Hsiu Chien (ORCID: https://orcid.org/0000-0001-8802-5728)
- Craig M. Zaidman (ORCID: https://orcid.org/0000-0002-1255-5877)
- Raquel Escudero (ORCID: https://orcid.org/0000-0001-6275-3225)
- Giovanni Baranello (ORCID: https://orcid.org/0000-0003-4871-6692)
- Volker Straub (ORCID: https://orcid.org/0000-0001-9046-3540)
- Susana Quijano‐Roy (ORCID: https://orcid.org/0000-0002-8503-6111)
- Michelle A. Farrar (ORCID: https://orcid.org/0000-0002-4472-0902)
- Yuh‐Jyh Jong (ORCID: https://orcid.org/0000-0002-1735-1101)
- Vincent Laugel (ORCID: https://orcid.org/0000-0002-5782-6538)
- Liesbeth De Waele (ORCID: https://orcid.org/0000-0001-7126-575X)
- Eugenio Maria Mercuri (ORCID: https://orcid.org/0000-0002-9851-5365)
- Julia Seibert
- I. Alecu
- Basil T. Darras (ORCID: https://orcid.org/0000-0002-1536-8145)
- Teresa Moreno (ORCID: https://orcid.org/0000-0002-7482-1587)
- Hugh J. McMillan (ORCID: https://orcid.org/0000-0001-8927-2018)
- Francesco Muntoni (ORCID: https://orcid.org/0000-0002-9102-5232)
- F. Freischläger
Institutions
- Università Cattolica del Sacro Cuore (IT)
- Novartis (Switzerland) (CH)
- Boston Children's Hospital (US)
- Harvard University (US)
- National Yang Ming Chiao Tung University (TW)
- Kaohsiung Medical University (TW)
- University of Ottawa (CA)
- Inserm (FR)
- Université de Versailles Saint-Quentin-en-Yvelines (FR)
- Washington University in St. Louis (US)
- Children's Hospital of Eastern Ontario (CA)
- Great Ormond Street Hospital (GB)
- Kaohsiung Medical University Chung-Ho Memorial Hospital (TW)
- UNSW Sydney (AU)
- Assistance Publique – Hôpitaux de Paris (FR)
- Centro Clinico Nemo (IT)
- Centro Hospitalar Lisboa Norte (PT)
- Novartis (United States) (US)
- Sydney Children's Hospital (AU)
- Tisch Hospital (US)
- Handicap neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie appliquées (FR)
- National Taiwan University Hospital (TW)
- Hôpital Raymond-Poincaré (FR)
- Hôpital d'Hautepierre (FR)
- Tris Pharma (United States) (US)
- University College London (GB)
- Newcastle University (GB)
- KU Leuven (BE)
Publication Details
- Journal
- Neurology
- Published
- 2024-12-30
- DOI
- https://doi.org/10.1212/wnl.0000000000210268
- Citations
- 15
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 6.57
Funders
- Pfizer
- Sanofi
- Biogen
- Alexion Pharmaceuticals
- Regeneron Pharmaceuticals
- PTC Therapeutics
- AveXis
- Sarepta Therapeutics
- Amicus Therapeutics
- European Commission
- Institut National de la Santé et de la Recherche Médicale
- Chugai Pharmaceutical