Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States

Background: Delandistrogene moxeparvovec (SRP-9001) is an investigational gene therapy that may delay progression of Duchenne muscular dystrophy (DMD), a severe, rare neuromuscular disease caused by DMD gene mutations. Early cost-effectiveness analyses are important to help contextualize the value o...

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Detalles Bibliográficos
Autores principales: Klimchak, Alexa C., Sedita, Lauren E., Rodino-Klapac, Louise R., Mendell, Jerry R., McDonald, Craig M., Gooch, Katherine L., Malone, Daniel C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Routledge 2023
Materias:
Original Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10228300/
https://www.ncbi.nlm.nih.gov/pubmed/37261034
http://dx.doi.org/10.1080/20016689.2023.2216518

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