spinal muscular atrophy type I
Rare disease · 2020–2020
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
What the PBS pays
$1M in government benefit over 2024–25, across 15 services.
This is spend on the medicines considered for this condition, not spend on the disease. A medicine used for several conditions is counted in full under each, so figures across conditions add to more than national PBS expenditure.
| Medicine | Government benefit |
|---|---|
| onasemnogene abeparvovec | $1.5M |
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.