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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Detalhes bibliográficos
Publicado no:J Mark Access Health Policy
Main Authors: Malone, Daniel C., Dean, Rebecca, Arjunji, Ramesh, Jensen, Ivar, Cyr, Phil, Miller, Beckley, Maru, Benit, Sproule, Douglas M., Feltner, Douglas E., Dabbous, Omar
Formato: Artigo
Idioma:Inglês
Publicado em: Routledge 2019
Assuntos:
Original Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6508058/
https://ncbi.nlm.nih.gov/pubmed/31105909
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/20016689.2019.1601484
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