spinal muscular atrophy type 1
Rare disease · 2021–2021
2decisions
1medicine
2carried consumer input
24individual 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. | Sep 2021 | Deferred | 12 individuals Spinal Muscular Atrophy Australia · Muscular Dystrophy Association |
| onasemnogene abeparvovec Treatment of spinal muscular atrophy Type I in patients under 2 years of age with bi-allelic mutations in the SMN1 gene. | May 2021 | 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: 2 of 2 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.