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

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