Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial
Year: 2025
Type: article
Language: en
Topic: Neurogenetic and Muscular Disorders Research
Authors and affiliations:
raw_author_name: Jennifer M. Kwon
raw_affiliation_strings: American Family Children's Hospital, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA. kwon@neurology.wisc.edu
raw_author_name: Francina Munell
raw_affiliation_strings: Department of Pediatric Neurology, Hospital Universitari Vall d'Hebron, Barcelona, Spain
raw_author_name: Laure Le Goff
raw_affiliation_strings: Department of Physical Medicine and Rehabilitation, Hôpital Femme Mère Enfant - Hospices Civils de Lyon, Lyon, France
raw_author_name: Kotaro Yuge
raw_affiliation_strings: Department of Pediatrics and Child Health, Kurume University School of Medicine, Kurume, Japan
raw_author_name: Tamaki Kato
raw_affiliation_strings: Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan
raw_author_name: Claude Cances
raw_affiliation_strings: Department of Paediatric Neurology, Reference Centre for Neuromuscular Disorders AOC, and Paediatric Clinical Research Unit Children's Hospital, Toulouse University Hospital Center, Toulouse, France
raw_author_name: Liesbeth De Waele
raw_affiliation_strings: Department of Development and Regeneration, KU Leuven, Leuven, Belgium
Department of Paediatric Neurology, Neuromuscular Reference Centre for Children, University Hospitals Leuven, Leuven, Belgium
raw_author_name: Ian R. Woodcock
raw_affiliation_strings: Department of Neurology, The Royal Children's Hospital, Melbourne, Victoria, Australia
The Murdoch Children's Research Institute, Parkville, Victoria, Australia
raw_author_name: Eugenio M. Mercuri
raw_affiliation_strings: Nemo Clinical Centre, Fondazione Policlinico Gemelli, IRCCS, Rome, Italy
Paediatric Neurology, Catholic University, Rome, Italy
raw_author_name: Crystal M. Proud
raw_affiliation_strings: Children's Hospital of The King's Daughters, Norfolk, VA, USA
raw_author_name: Basil T. Darras
raw_affiliation_strings: Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA
raw_author_name: Leslie H. Hayes
raw_affiliation_strings: Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA
raw_author_name: Maryam Oskoui
raw_affiliation_strings: Centre for Outcomes Research and Evaluation, Research Institute of the McGill University Health Centre, Montreal, Quebec, Canada
Departments of Neurology and Neurosurgery and Pediatric, McGill University, Montreal, Quebec, Canada
raw_author_name: Jeannie Visootsak
raw_affiliation_strings: Novartis Pharmaceuticals, East Hanover, NJ, USA
raw_author_name: Gemma Williams
raw_affiliation_strings: Novartis Pharmaceuticals, London, UK
raw_author_name: Andreja Ilić
raw_affiliation_strings: Novartis Pharmaceuticals, Basel, Switzerland
raw_author_name: Lina Yang
raw_affiliation_strings: Novartis Pharmaceuticals, East Hanover, NJ, USA
raw_author_name: W. Ludo van der Pol
raw_affiliation_strings: UMC Utrecht Brain Center, Department of Neurology and Neurosurgery, University Medical Center Utrecht, Utrecht, The Netherlands