[Nusinersen in spinal muscular atrophy]

Gonzalez L, Pichon-Riviere A, Augustovski F, García Martí S, Alcaraz A, Bardach A, Ciapponi A.
Record ID 32018001406
Spanish
Original Title: Nusinersen en atrofia muscular espinal
Authors' recommendations: CONCLUSIONS High-quality evidence from only one randomized clinical trial shows that the intrathecal use of nusinersen in patients under seven month old, with Type I spinal muscular atrophy decreased mortality and the need for permanent mechanical ventilation. In these patients, as well as in those with subtypes II and III, it also proved to develop and improve motor milestones (sitting, standing, walking, etc.). No studies assessing the effect of nusinersen on mortality and need for mechanical ventilation in subtype II and III patients have been found. In our country, it has not been authorized by ANMAT (Argentine National Administration of Drugs, Foods, and Medical Devices), and it is considered a drug for compassive use. Although expensive, its cost-efficiency is unknown, thus its estimated financial impact is very high. Only some United States private health sponsors consider its coverage.
Details
Project Status: Completed
Year Published: 2018
URL for published report: https://www.iecs.org.ar/home-ets/
English language abstract: An English language summary is available
Publication Type: Not Assigned
Country: Argentina
MeSH Terms
  • Muscular Atrophy, Spinal
  • Oligonucleotides
  • Oligonucleotides, Antisense
  • Drug Therapy
Contact
Organisation Name: Institute for Clinical Effectiveness and Health Policy
Contact Address: Dr. Emilio Ravignani 2024, Buenos Aires - Argentina, C1414 CABA
Contact Name: info@iecs.org.ar
Contact Email: info@iecs.org.ar
Copyright: Institute for Clinical Effectiveness and Health Policy (IECS)
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.