Onasemnogene abeparvovec gene therapy for treatment of patients with spinal muscular atrophy: Updated real-world practical considerations.
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| Title: | Onasemnogene abeparvovec gene therapy for treatment of patients with spinal muscular atrophy: Updated real-world practical considerations. |
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| Authors: | Proud CM; Children's Hospital of The King's Daughters, Norfolk, VA, USA., Kichula EA; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Matesanz SE; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.; University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA., Kumar A; Division of Pediatric Neurology, Penn State Health Children's Hospital, Hershey, PA, USA., Saito K; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan., Laverty CG; Department of Neurosciences, University of California, San Diego, San Diego, CA, USA., Farrar MA; School of Clinical Medicine, UNSW Medicine and Health, UNSW Sydney, Sydney, NSW, Australia.; Department of Neurology, Sydney Children's Hospital Network, Sydney, NSW, Australia., Bharucha-Goebel DX; Division of Neurology, Children's National Hospital, Washington, DC, USA.; Jerry R. Mendell Center for Gene Therapy and Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Haberlová J; Neuromuscular Center, Department of Pediatric Neurology, Faculty Hospital Motol, 2nd School of Medicine Charles University, Prague, Czech Republic., Mundada V; Department of Paediatric Neuroscience, Aster DM Healthcare, Medcare Hospitals, Dubai, UAE., Kwon JM; School of Medicine and Public Health, University of Wisconsin, Madison, WI, USA., McMillan HJ; Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada. |
| Source: | Journal of neuromuscular diseases [J Neuromuscul Dis] 2026 Jul; Vol. 13 (4), pp. 587-601. Date of Electronic Publication: 2025 Nov 10. |
| Publication Type: | Journal Article; Review |
| Journal Info: | Publisher: IOS Press Country of Publication: United States NLM ID: 101649948 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2214-3602 (Electronic) Linking ISSN: 22143599 NLM ISO Abbreviation: J Neuromuscul Dis Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 2214-3602 |
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| DOI: | 10.1177/22143602251391258 |
