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Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR).
Zaidman, Craig M; Proud, Crystal M; McDonald, Craig M; Lehman, Kelly J; Goedeker, Natalie L; Mason, Stefanie; Murphy, Alexander P; Guridi, Maitea; Wang, Shufang; Reid, Carol; Darton, Eddie; Wandel, Christoph; Lewis, Sarah; Malhotra, Jyoti; Griffin, Danielle A; Potter, Rachael A; Rodino-Klapac, Louise R; Mendell, Jerry R.
Afiliación
  • Zaidman CM; Department of Neurology, Washington University in St. Louis, St. Louis, MO, USA.
  • Proud CM; Children's Hospital of the King's Daughters, Norfolk, VA, USA.
  • McDonald CM; University of California Davis Health, Sacramento, CA, USA.
  • Lehman KJ; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
  • Goedeker NL; Department of Neurology, Washington University in St. Louis, St. Louis, MO, USA.
  • Mason S; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Murphy AP; Roche Products Ltd, Welwyn Garden City, UK.
  • Guridi M; F. Hoffmann-La Roche Ltd, Basel, Switzerland.
  • Wang S; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Reid C; Roche Products Ltd, Welwyn Garden City, UK.
  • Darton E; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Wandel C; F. Hoffmann-La Roche Ltd, Basel, Switzerland.
  • Lewis S; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Malhotra J; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Griffin DA; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Potter RA; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Rodino-Klapac LR; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Mendell JR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
Ann Neurol ; 94(5): 955-968, 2023 11.
Article en En | MEDLINE | ID: mdl-37539981
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Distrofia Muscular de Duchenne Límite: Humans / Male Idioma: En Revista: Ann Neurol Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Distrofia Muscular de Duchenne Límite: Humans / Male Idioma: En Revista: Ann Neurol Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos