Real-world outcomes of delandistrogene moxeparvovec gene therapy: Motor outcomes and emerging safety concerns.
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| Title: | Real-world outcomes of delandistrogene moxeparvovec gene therapy: Motor outcomes and emerging safety concerns. |
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| Authors: | Keselman DJ; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Neurology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA., Small JC; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Seneviratne T; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., McCague S; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Kaschak TL; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Yum SW; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Neurology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA., O'Brien A; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Brandsema JF; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Neurology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA., Diamond T; Bioinformatics and Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA., Loomes KM; Bioinformatics and Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA., Lin KY; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Cardiology, Cardiac Center, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Wittlieb-Weber CA; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Cardiology, Cardiac Center, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Mayer OH; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Pulmonology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Daniel SG; Bioinformatics and Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Waldman AT; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Neurology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Samelson-Jones BJ; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Matesanz SE; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Neurology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. Electronic address: matesanzs@chop.edu., George LA; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. Electronic address: georgel@chop.edu. |
| Source: | Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2026 Jan 07; Vol. 34 (1), pp. 123-130. Date of Electronic Publication: 2025 Oct 04. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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