Spinraza and Zolgensma for spinal muscular atrophy: effectiveness and value : final evidence report

Spinraza and Zolgensma for spinal muscular atrophy: effectiveness and value : final evidence report

Collection:
Health Policy and Services Research
Author(s):
Ellis, Alexandra, author
Mickle, Kristin, author
Herron-Smith, Serina, author
Kumar, Varun M., author
Cianciolo, Laura, author
Seidner, Matt, author
Rind, David, author
Pearson, Steven, author
Thokala, P. (Praveen), author
Stevenson, M. (Matt), author
Contributor(s):
Institute for Clinical and Economic Review, issuing body.
Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, May 24, 2019
Language(s):
English
Format:
Text
Subject(s):
Genetic Therapy
Muscular Atrophy, Spinal -- drug therapy
United States
Genre(s):
Technical Report
Abstract:
Because of heightened interest in Zolgensma in light of its FDA approval and the limited evidence base at the time of publication of ICER’s Final Report, we are including this brief discussion of additional data/interim analyses from ongoing trials of Zolgensma that have been made public through conferences (Muscular Dystrophy Association Clinical and Scientific Conference April 13- 17, 2019 and American Academy of Neurology Annual Meeting May 4-10, 2019)1-3 and manufacturer press releases. This is not a systematic review of new evidence. Note that outside of this text box, no other sections of the report have been revised.
Copyright:
Reproduced with permission of the copyright holder. Further use of the material is subject to CC BY license. (More information)
Extent:
1 online resource (1 PDF file (various pagings))
Illustrations:
Illustrations
NLM Unique ID:
9918402188606676 (See catalog record)
Permanent Link:
http://resource.nlm.nih.gov/9918402188606676