SMA with 3 copies SMN2 gene
Neurology · 2023–2023
1decision
1medicine
1carried consumer input
1individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| nusinersen Initial treatment of pre-symptomatic spinal muscular atrophy (SMA) in individuals with SMN1 deletion or mutation and 3 copies of the SMN2 gene, aged less than… | Jul 2023 | Recommended with restriction | 1 individuals Muscular Dystrophy Association |
Who spoke
Muscular Dystrophy Association
Conditions are classified by a language model from the indication text of each submission, so an individual label here can be wrong. Consumer input is read from the document's own consumer comments section: 1 of 1 decisions since 2014 carried some. Silence means no comment reached the committee through that facility, not that nobody was affected. More on how this was built.