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Expert Opinion on Biological Therapy Volume 22, 2022 - Issue 9: Gene therapy
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Drug Evaluation

Onasemnogene abeparvovec for the treatment of spinal muscular atrophy

Hugh J. McMillana Departments of Pediatrics, Neurology & Neurosurgery, Montreal Children’s Hospital, McGill University Health Centre, Montreal, CanadaCorrespondence[email protected]
ORCID Iconhttps://orcid.org/0000-0001-8927-2018View further author information
ORCID Icon,
Crystal M. Proudb Children’s Hospital of the King’s Daughters, Norfolk, VA, United StatesView further author information
,
Michelle A. Farrarc School of Clinical Medicine, UNSW Medicine and Health, UNSW Sydney, Sydney, NSW, Australia;d Department of Neurology, Sydney Children’s Hospital Network, Sydney, NSW, AustraliaView further author information
,
Ian E. Alexandere Gene Therapy Research Unit, Children’s Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children’s Hospitals Network, Westmead, NSW, Australia;f Discipline of Child and Adolescent Health, Sydney Medical School, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW, AustraliaView further author information
,
Francesco Muntonig The Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health London, UK;h NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, London, UKView further author information
&
Laurent Servaisi Department of Pediatrics, Centre Hospitalier Universitaire de Liège & Université de Liège, Liège, Belgium;j MDUK Oxford Neuromuscular Centre, University of Oxford, Oxford, UKORCID Iconhttps://orcid.org/0000-0001-9270-4061View further author information
ORCID Icon
Pages 1075-1090 | Received 12 Jan 2022, Accepted 12 Apr 2022, Published online: 02 May 2022

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