spinal muscular atrophy type I
Rare disease · 2020–2020
1decision
1medicine
1carried consumer input
12individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| onasemnogene abeparvovec Treatment of spinal muscular atrophy (SMA) Type I in patients under 2 years of age with bi-allelic mutations in the SMN1 gene. | Nov 2020 | Deferred | 12 individuals Spinal Muscular Atrophy Australia · Muscular Dystrophy Association |
Who spoke
Spinal Muscular Atrophy Australia · 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.