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Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial.
Mendell, Jerry R; Sahenk, Zarife; Lehman, Kelly J; Lowes, Linda P; Reash, Natalie F; Iammarino, Megan A; Alfano, Lindsay N; Lewis, Sarah; Church, Kathleen; Shell, Richard; Potter, Rachael A; Griffin, Danielle A; Hogan, Mark; Wang, Shufang; Mason, Stefanie; Darton, Eddie; Rodino-Klapac, Louise R.
Afiliación
  • Mendell JR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Sahenk Z; Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
  • Lehman KJ; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Lowes LP; Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
  • Reash NF; Departments of Pathology and Neurology, The Ohio State University, Columbus, Ohio, USA.
  • Iammarino MA; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Alfano LN; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Lewis S; Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
  • Church K; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Shell R; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Potter RA; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Griffin DA; Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.
  • Hogan M; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Wang S; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Mason S; Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.
  • Darton E; Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.
  • Rodino-Klapac LR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
Muscle Nerve ; 69(1): 93-98, 2024 Jan.
Article en En | MEDLINE | ID: mdl-37577753
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Distrofia Muscular de Duchenne Idioma: En Revista: Muscle Nerve Año: 2024 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Base de datos: MEDLINE Asunto principal: Distrofia Muscular de Duchenne Idioma: En Revista: Muscle Nerve Año: 2024 Tipo del documento: Article