Niemann-Pick disease Type C
Rare disease · 2011–2011
1decision
1medicine
0carried consumer input
0individual submissions
Every decision here pre-dates the consumer comments facility, so no input would be recorded either way.
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| miglustat Treatment of progressive neurological manifestations in adults and paediatric patients with Niemann-Pick disease Type C (NP-C) disease. | Nov 2011 | Not recommended | unclear |
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: 0 of 0 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.