Intrathecal onasemnogene abeparvovec (OAV101 IT) for treatment-experienced patients with spinal muscular atrophy (SMA): Phase 3B, open-label strength study
Year: 2025
Type: article
Language: en
Topic: Neurogenetic and Muscular Disorders Research
Authors and affiliations:
raw_author_name: Jennifer Kwon
raw_affiliation_strings: American Family Children's Hospital, University of Wisconsin School of Medicine and Public Health, Department of Neurology, Madison, United States of America
raw_author_name: Francina Munell
raw_affiliation_strings: Hospital Vall d'Hebron, Department of Neurology, Barcelona, Spain
raw_author_name: Laure Le Goff
raw_affiliation_strings: Hôpital Femme Mère Enfant - Hospices Civils de Lyon, N/a, Lyon, France
raw_author_name: Kotaro Yuge
raw_affiliation_strings: Kurume University Hospital, Department of Pediatrics and Child Health, Kurume, Japan
raw_author_name: Tamaki Kato
raw_affiliation_strings: Hospital Clinical Genomics, Tokyo Women's Medical University, N/a, Tokyo, Japan
raw_author_name: Claude Cances
raw_affiliation_strings: Reference Centre for Neuromuscular Disorders AOC, and Paediatric Clinical Research Unit Children's Hospital, Toulouse University Hospital Center, Department of Paediatric Neurology, Toulouse, France
raw_author_name: Liesbeth De Waele
raw_affiliation_strings: University Hospitals Leuven, Department of Paediatric Neurology, Leuven, Belgium
raw_author_name: Ian Woodcock
raw_affiliation_strings: The Murdoch Children's Research Institute, N/a, Parkville, Australia
The Royal Children's Hospital, Department of Neurology, Melbourne, Australia
raw_author_name: Eugenio Mercuri
raw_affiliation_strings: Catholic University, Department of Paediatric Neurology and Nemo Clinical Centre, Rome, Italy
raw_author_name: Crystal Proud
raw_affiliation_strings: Children's Hospital of the King's Daughters, N/a, Norfolk, United States of America
raw_author_name: Basil Darras
raw_affiliation_strings: Boston Children's Hospital, Harvard Medical School, Department of Neurology, Boston, United States of America
raw_author_name: Leslie Hayes
raw_affiliation_strings: Boston Children's Hospital, Harvard Medical School, Department of Neurology, Boston, United States of America
raw_author_name: Maryam Oskoui
raw_affiliation_strings: McGill University Health Centre, Departments of Neurology & Neurosurgery and Paediatric, Montréal, Canada
Montreal Children's Hospital, Centre for Outcomes Research and Evaluation, Montréal, Canada
raw_author_name: Jeannie Visootsak
raw_affiliation_strings: Novartis Pharmaceuticals, Neuroscience and Gene Therapies, East Hanover, United States of America
raw_author_name: Gemma Williams
raw_affiliation_strings: Novartis Pharmaceuticals, N/a, London, United Kingdom
raw_author_name: Anna Lee
raw_affiliation_strings: Novartis Pharmaceuticals, Neuroscience and Gene Therapies, East Hanover, United States of America
raw_author_name: Andreja Ilić
raw_affiliation_strings: Novartis Pharmaceuticals, Neuroscience R&d, Basel, Switzerland
raw_author_name: Lina Yang
raw_affiliation_strings: Novartis Pharmaceuticals, N/a, East Hanover, United States of America
raw_author_name: W. Ludo Van Der Pol
raw_affiliation_strings: University Medical Center Utrecht, Division Neurosciences, Department of Neurology and Neurosurgery, Neuromuscular Disorders, Utrecht, the Netherlands