Disease index

Niemann-Pick Type C disease

Rare disease · 2010–2010

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

MedicineMeetingOutcomeConsumer input
miglustat Treatment of progressive neurological manifestations in adult and paediatric patients with Niemann-Pick Type C disease. Jul 2010 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.