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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.
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
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  Data: Real-world outcomes of delandistrogene moxeparvovec gene therapy: Motor outcomes and emerging safety concerns.
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  Data: <searchLink fieldCode="AU" term="%22Keselman+DJ%22">Keselman DJ</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Small+JC%22">Small JC</searchLink>; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22Seneviratne+T%22">Seneviratne T</searchLink>; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22McCague+S%22">McCague S</searchLink>; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22Kaschak+TL%22">Kaschak TL</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22Yum+SW%22">Yum SW</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22O'Brien+A%22">O'Brien A</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22Brandsema+JF%22">Brandsema JF</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Diamond+T%22">Diamond T</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Loomes+KM%22">Loomes KM</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Lin+KY%22">Lin KY</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Wittlieb-Weber+CA%22">Wittlieb-Weber CA</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Mayer+OH%22">Mayer OH</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Daniel+SG%22">Daniel SG</searchLink>; Bioinformatics and Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22Waldman+AT%22">Waldman AT</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Samelson-Jones+BJ%22">Samelson-Jones BJ</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Matesanz+SE%22">Matesanz SE</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22George+LA%22">George LA</searchLink>; 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.
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