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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.
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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  Data: Onasemnogene abeparvovec gene therapy for treatment of patients with spinal muscular atrophy: Updated real-world practical considerations.
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  Data: <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="%22Kichula+EA%22">Kichula EA</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Matesanz+SE%22">Matesanz SE</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.; University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Kumar+A%22">Kumar A</searchLink>; Division of Pediatric Neurology, Penn State Health Children's Hospital, Hershey, PA, USA.<br /><searchLink fieldCode="AU" term="%22Saito+K%22">Saito K</searchLink>; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.<br /><searchLink fieldCode="AU" term="%22Laverty+CG%22">Laverty CG</searchLink>; Department of Neurosciences, University of California, San Diego, San Diego, CA, USA.<br /><searchLink fieldCode="AU" term="%22Farrar+MA%22">Farrar MA</searchLink>; School of Clinical Medicine, UNSW Medicine and Health, UNSW Sydney, Sydney, NSW, Australia.; Department of Neurology, Sydney Children's Hospital Network, Sydney, NSW, Australia.<br /><searchLink fieldCode="AU" term="%22Bharucha-Goebel+DX%22">Bharucha-Goebel DX</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Haberlová+J%22">Haberlová J</searchLink>; Neuromuscular Center, Department of Pediatric Neurology, Faculty Hospital Motol, 2nd School of Medicine Charles University, Prague, Czech Republic.<br /><searchLink fieldCode="AU" term="%22Mundada+V%22">Mundada V</searchLink>; Department of Paediatric Neuroscience, Aster DM Healthcare, Medcare Hospitals, Dubai, UAE.<br /><searchLink fieldCode="AU" term="%22Kwon+JM%22">Kwon JM</searchLink>; School of Medicine and Public Health, University of Wisconsin, Madison, WI, USA.<br /><searchLink fieldCode="AU" term="%22McMillan+HJ%22">McMillan HJ</searchLink>; Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.
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  Data: <searchLink fieldCode="JN" term="%22101649948%22">Journal of neuromuscular diseases</searchLink> [J Neuromuscul Dis] 2026 Jul; Vol. 13 (4), pp. 587-601. <i>Date of Electronic Publication: </i>2025 Nov 10.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22IOS+Press%22">IOS Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>101649948 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>2214-3602 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2222143599%22">22143599 </searchLink><i>NLM ISO Abbreviation: </i>J Neuromuscul Dis <i>Subsets: </i>MEDLINE
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        Value: 10.1177/22143602251391258
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              Text: 2026 Jul
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