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
English language abstract: An English language summary is available
Publication Type: Full HTA
Country: Scotland
MeSH Terms
  • Spinal Muscular Atrophies of Childhood
  • Genetic Therapy
  • Biological Products
  • Recombinant Fusion Proteins
  • Child
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.