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Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients

Background: Spinal muscular atrophy type 1 (SMA1) is a devastating genetic disease for which gene-replacement therapy may bring substantial survival and quality of life benefits. Objective: This study investigated the cost-effectiveness of onasemnogene abeparvovec (AVXS-101) gene-replacement therapy...

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Detalles Bibliográficos
Autores principales:	Malone, Daniel C., Dean, Rebecca, Arjunji, Ramesh, Jensen, Ivar, Cyr, Phil, Miller, Beckley, Maru, Benit, Sproule, Douglas M., Feltner, Douglas E., Dabbous, Omar
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Routledge 2019
Materias:	Original Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6508058/ https://www.ncbi.nlm.nih.gov/pubmed/31105909 http://dx.doi.org/10.1080/20016689.2019.1601484

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