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Cardiotoxic Effects and Microdystrophin Expression after Gene Therapy for DMD.

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Title: Cardiotoxic Effects and Microdystrophin Expression after Gene Therapy for DMD.
Authors: Samelson-Jones BJ; Children's Hospital of Philadelphia, Philadelphia., Martos-Rus C; Children's Hospital of Philadelphia, Philadelphia., Yrigollen CM; Children's Hospital of Philadelphia, Philadelphia., Tecedor L; Children's Hospital of Philadelphia, Philadelphia., Small JC; Children's Hospital of Philadelphia, Philadelphia., Matesanz SE; Children's Hospital of Philadelphia, Philadelphia., Watson CT; Children's Hospital of Philadelphia, Philadelphia., Vanden Heuvel AR; Children's Hospital of Philadelphia, Philadelphia., Carrig S; Children's Hospital of Philadelphia, Philadelphia., Yum SW; Children's Hospital of Philadelphia, Philadelphia., Brandsema JF; Children's Hospital of Philadelphia, Philadelphia., Lin K; Children's Hospital of Philadelphia, Philadelphia., Wittlieb-Weber C; Children's Hospital of Philadelphia, Philadelphia., Viaene AN; Children's Hospital of Philadelphia, Philadelphia., Russo PP; Children's Hospital of Philadelphia, Philadelphia., Partington S; University of Pennsylvania, Philadelphia., Huynh K; Children's Hospital of Philadelphia, Philadelphia., Seneviratne T; Children's Hospital of Philadelphia, Philadelphia., Juarez Rojas S; Emory University School of Medicine, Atlanta., Doshi BS; Emory University School of Medicine, Atlanta., Shieh P; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles., Flanigan KM; Nationwide Children's Hospital, Columbus, OH., Frair E; Nationwide Children's Hospital, Columbus, OH., Nicolau S; Nationwide Children's Hospital, Columbus, OH., Lawlor MW; Diverge Translational Science Laboratory, Milwaukee., Crudele JM; University of Washington School of Medicine, Seattle., Davidson BL; Children's Hospital of Philadelphia, Philadelphia., George LA; Children's Hospital of Philadelphia, Philadelphia.
Source: The New England journal of medicine [N Engl J Med] 2026 Aug 06; Vol. 395 (6), pp. 615-617. Date of Electronic Publication: 2026 Jul 22.
Publication Type: Letter
Journal Info: Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE; In Process
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  Data: <searchLink fieldCode="AU" term="%22Samelson-Jones+BJ%22">Samelson-Jones BJ</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Martos-Rus+C%22">Martos-Rus C</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Yrigollen+CM%22">Yrigollen CM</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Tecedor+L%22">Tecedor L</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Small+JC%22">Small JC</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Matesanz+SE%22">Matesanz SE</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Watson+CT%22">Watson CT</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Vanden+Heuvel+AR%22">Vanden Heuvel AR</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Carrig+S%22">Carrig S</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Yum+SW%22">Yum SW</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Brandsema+JF%22">Brandsema JF</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Lin+K%22">Lin K</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Wittlieb-Weber+C%22">Wittlieb-Weber C</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Viaene+AN%22">Viaene AN</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Russo+PP%22">Russo PP</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Partington+S%22">Partington S</searchLink>; University of Pennsylvania, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Huynh+K%22">Huynh K</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Seneviratne+T%22">Seneviratne T</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22Juarez+Rojas+S%22">Juarez Rojas S</searchLink>; Emory University School of Medicine, Atlanta.<br /><searchLink fieldCode="AU" term="%22Doshi+BS%22">Doshi BS</searchLink>; Emory University School of Medicine, Atlanta.<br /><searchLink fieldCode="AU" term="%22Shieh+P%22">Shieh P</searchLink>; David Geffen School of Medicine, University of California, Los Angeles, Los Angeles.<br /><searchLink fieldCode="AU" term="%22Flanigan+KM%22">Flanigan KM</searchLink>; Nationwide Children's Hospital, Columbus, OH.<br /><searchLink fieldCode="AU" term="%22Frair+E%22">Frair E</searchLink>; Nationwide Children's Hospital, Columbus, OH.<br /><searchLink fieldCode="AU" term="%22Nicolau+S%22">Nicolau S</searchLink>; Nationwide Children's Hospital, Columbus, OH.<br /><searchLink fieldCode="AU" term="%22Lawlor+MW%22">Lawlor MW</searchLink>; Diverge Translational Science Laboratory, Milwaukee.<br /><searchLink fieldCode="AU" term="%22Crudele+JM%22">Crudele JM</searchLink>; University of Washington School of Medicine, Seattle.<br /><searchLink fieldCode="AU" term="%22Davidson+BL%22">Davidson BL</searchLink>; Children's Hospital of Philadelphia, Philadelphia.<br /><searchLink fieldCode="AU" term="%22George+LA%22">George LA</searchLink>; Children's Hospital of Philadelphia, Philadelphia.
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  Data: <searchLink fieldCode="JN" term="%220255562%22">The New England journal of medicine</searchLink> [N Engl J Med] 2026 Aug 06; Vol. 395 (6), pp. 615-617. <i>Date of Electronic Publication: </i>2026 Jul 22.
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