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