Onasemnogene abeparvovec treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene
SMC
Record ID 32018001093
English
Authors' objectives:
To assess clinical and cost effectiveness.
Details
Project Status:
Completed
Year Published:
2021
URL for published report:
https://www.scottishmedicines.org.uk/medicines-advice/onasemnogene-abeparvovec-zolgensma-full-smc2311/
English language abstract:
An English language summary is available
Publication Type:
Full HTA
Country:
Scotland, United Kingdom
MeSH Terms
- Spinal Muscular Atrophies of Childhood
- Genetic Therapy
- Biological Products
- Recombinant Fusion Proteins
Contact
Organisation Name:
Scottish Medicines Consortium (SMC)
Contact Address:
Delta House | 50 West Nile Street
Contact Name:
Susan Downie
Contact Email:
susan.downie3@nhs.scot
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.