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

• Principais autores: Daniel C. Malone (Autor), Rebecca Dean (Autor), Ramesh Arjunji (Autor), Ivar Jensen (Autor), Phil Cyr (Autor), Beckley Miller (Autor), Benit Maru (Autor), Douglas M. Sproule (Autor), Douglas E. Feltner (Autor), Omar Dabbous (Autor)
• Formato: Livro
• Publicado em: Taylor & Francis Group, 2019-01-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

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...