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Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States.
Klimchak, Alexa C; Sedita, Lauren E; Rodino-Klapac, Louise R; Mendell, Jerry R; McDonald, Craig M; Gooch, Katherine L; Malone, Daniel C.
Afiliação
  • Klimchak AC; Global HEOR, RWE & Analytics, Sarepta Therapeutics, Inc, Cambridge, MA, USA.
  • Sedita LE; Global HEOR, RWE & Analytics, Sarepta Therapeutics, Inc, Cambridge, MA, USA.
  • Rodino-Klapac LR; Global HEOR, RWE & Analytics, Sarepta Therapeutics, Inc, Cambridge, MA, USA.
  • Mendell JR; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
  • McDonald CM; Department of Pediatrics and Neurology, The Ohio State University, Columbus, OH, USA.
  • Gooch KL; Department of Pediatrics, University of California Davis School of Medicine, Davis, CA, USA.
  • Malone DC; Research & Development, Sarepta Therapeutics, Inc, Cambridge, MA, USA.
J Mark Access Health Policy ; 11(1): 2216518, 2023.
Article em En | MEDLINE | ID: mdl-37261034
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Ano de publicação: 2023 Tipo de documento: Article