acid sphingomyelinase deficiency
Rare disease · 2023–2023
1decision
1medicine
1carried consumer input
3individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| olipudase alfa Treatment of acid sphingomyelinase deficiency (ASMD) type A/B or type B in paediatric and adult patients. Olipudase alfa is an enzyme replacement therapy… | Jul 2023 | Not recommended | 3 individuals |
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.