[Nusinersen (Spinraza®) in spinal muscular atrophy]
Gonzalez L, Pichon-Riviere A, Augustovski F, García Martí S, Alcaraz A, Bardach A, Ciapponi A
Record ID 32017000306
Spanish
Authors' recommendations:
Evidence from partial results of one randomized clinical trial still unpublished shows that the intrathecal use of nusinersen in patients with type I spinal muscular atrophy, under seven months, might reduce mortality and the need for continuous ventilatory support. It may also improve developing and acquiring certain capacities (including sitting down, standing, walking). Evidence from non-controlled studies which assessed its use in patient with other subtypes of this condition, showed similar results in motor function.
In our country, the National Administration of Drugs, Foods and Medical Devices (ANMAT) has not approved it and is considered a drug for compassionate use. It is expensive and its cost-effectiveness is unknown. Only some United States private health sponsors consider its coverage.
Details
Project Status:
Completed
Year Published:
2017
URL for published report:
www.iecs.org.ar/home-ets/
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
Argentina
MeSH Terms
- Humans
- Muscular Atrophy, Spinal
- Oligonucleotides
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:
<p>Institute for Clinical Effectiveness and Health Policy (IECS)</p>
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.