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

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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Bibliographic Details
Main Authors: Daniel C. Malone, Rebecca Dean, Ramesh Arjunji, Ivar Jensen, Phil Cyr, Beckley Miller, Benit Maru, Douglas M. Sproule, Douglas E. Feltner, Omar Dabbous
Format: Article
Language:English
Published: MDPI AG 2019-01-01
Series:Journal of Market Access & Health Policy
Subjects:
avxs-101
cost-effectiveness
gene-replacement therapy
nusinersen
onasemnogene abeparvovec
spinal muscular atrophy type 1
Online Access:http://dx.doi.org/10.1080/20016689.2019.1601484

Internet

http://dx.doi.org/10.1080/20016689.2019.1601484

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