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Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.

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Title: Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
Authors: Kwon JM; American Family Children's Hospital, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA. kwon@neurology.wisc.edu., Munell F; Department of Pediatric Neurology, Hospital Universitari Vall d'Hebron, Barcelona, Spain., Le Goff L; Department of Physical Medicine and Rehabilitation, Hôpital Femme Mère Enfant - Hospices Civils de Lyon, Lyon, France., Yuge K; Department of Pediatrics and Child Health, Kurume University School of Medicine, Kurume, Japan., Kato T; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan., Cances C; Department of Paediatric Neurology, Reference Centre for Neuromuscular Disorders AOC, and Paediatric Clinical Research Unit Children's Hospital, Toulouse University Hospital Center, Toulouse, France., De Waele L; Department of Paediatric Neurology, Neuromuscular Reference Centre for Children, University Hospitals Leuven, Leuven, Belgium.; Department of Development and Regeneration, KU Leuven, Leuven, Belgium., Woodcock IR; Department of Neurology, The Royal Children's Hospital, Melbourne, Victoria, Australia.; The Murdoch Children's Research Institute, Parkville, Victoria, Australia., Mercuri EM; Paediatric Neurology, Catholic University, Rome, Italy.; Nemo Clinical Centre, Fondazione Policlinico Gemelli, IRCCS, Rome, Italy., Proud CM; Children's Hospital of The King's Daughters, Norfolk, VA, USA., Darras BT; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., Hayes LH; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., Oskoui M; Departments of Neurology and Neurosurgery and Pediatric, McGill University, Montreal, Quebec, Canada.; Centre for Outcomes Research and Evaluation, Research Institute of the McGill University Health Centre, Montreal, Quebec, Canada., Visootsak J; Novartis Pharmaceuticals, East Hanover, NJ, USA., Williams G; Novartis Pharmaceuticals, London, UK., Ilić A; Novartis Pharmaceuticals, Basel, Switzerland., Yang L; Novartis Pharmaceuticals, East Hanover, NJ, USA., van der Pol WL; UMC Utrecht Brain Center, Department of Neurology and Neurosurgery, University Medical Center Utrecht, Utrecht, The Netherlands.
Source: Nature medicine [Nat Med] 2026 Feb; Vol. 32 (2), pp. 488-493. Date of Electronic Publication: 2025 Dec 08.
Publication Type: Journal Article; Clinical Trial, Phase III; Multicenter Study
Journal Info: Publisher: Nature Publishing Company Country of Publication: United States NLM ID: 9502015 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1546-170X (Electronic) Linking ISSN: 10788956 NLM ISO Abbreviation: Nat Med Subsets: MEDLINE
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  Data: Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
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  Data: <searchLink fieldCode="AU" term="%22Kwon+JM%22">Kwon JM</searchLink>; American Family Children's Hospital, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA. kwon@neurology.wisc.edu.<br /><searchLink fieldCode="AU" term="%22Munell+F%22">Munell F</searchLink>; Department of Pediatric Neurology, Hospital Universitari Vall d'Hebron, Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22Le+Goff+L%22">Le Goff L</searchLink>; Department of Physical Medicine and Rehabilitation, Hôpital Femme Mère Enfant - Hospices Civils de Lyon, Lyon, France.<br /><searchLink fieldCode="AU" term="%22Yuge+K%22">Yuge K</searchLink>; Department of Pediatrics and Child Health, Kurume University School of Medicine, Kurume, Japan.<br /><searchLink fieldCode="AU" term="%22Kato+T%22">Kato T</searchLink>; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.<br /><searchLink fieldCode="AU" term="%22Cances+C%22">Cances C</searchLink>; Department of Paediatric Neurology, Reference Centre for Neuromuscular Disorders AOC, and Paediatric Clinical Research Unit Children's Hospital, Toulouse University Hospital Center, Toulouse, France.<br /><searchLink fieldCode="AU" term="%22De+Waele+L%22">De Waele L</searchLink>; Department of Paediatric Neurology, Neuromuscular Reference Centre for Children, University Hospitals Leuven, Leuven, Belgium.; Department of Development and Regeneration, KU Leuven, Leuven, Belgium.<br /><searchLink fieldCode="AU" term="%22Woodcock+IR%22">Woodcock IR</searchLink>; Department of Neurology, The Royal Children's Hospital, Melbourne, Victoria, Australia.; The Murdoch Children's Research Institute, Parkville, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Mercuri+EM%22">Mercuri EM</searchLink>; Paediatric Neurology, Catholic University, Rome, Italy.; Nemo Clinical Centre, Fondazione Policlinico Gemelli, IRCCS, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Proud+CM%22">Proud CM</searchLink>; Children's Hospital of The King's Daughters, Norfolk, VA, USA.<br /><searchLink fieldCode="AU" term="%22Darras+BT%22">Darras BT</searchLink>; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Hayes+LH%22">Hayes LH</searchLink>; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Oskoui+M%22">Oskoui M</searchLink>; Departments of Neurology and Neurosurgery and Pediatric, McGill University, Montreal, Quebec, Canada.; Centre for Outcomes Research and Evaluation, Research Institute of the McGill University Health Centre, Montreal, Quebec, Canada.<br /><searchLink fieldCode="AU" term="%22Visootsak+J%22">Visootsak J</searchLink>; Novartis Pharmaceuticals, East Hanover, NJ, USA.<br /><searchLink fieldCode="AU" term="%22Williams+G%22">Williams G</searchLink>; Novartis Pharmaceuticals, London, UK.<br /><searchLink fieldCode="AU" term="%22Ilić+A%22">Ilić A</searchLink>; Novartis Pharmaceuticals, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Yang+L%22">Yang L</searchLink>; Novartis Pharmaceuticals, East Hanover, NJ, USA.<br /><searchLink fieldCode="AU" term="%22van+der+Pol+WL%22">van der Pol WL</searchLink>; UMC Utrecht Brain Center, Department of Neurology and Neurosurgery, University Medical Center Utrecht, Utrecht, The Netherlands.
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  Data: <searchLink fieldCode="JN" term="%229502015%22">Nature medicine</searchLink> [Nat Med] 2026 Feb; Vol. 32 (2), pp. 488-493. <i>Date of Electronic Publication: </i>2025 Dec 08.
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