type 1 Gaucher disease
Rare disease · 2008–2014
2decisions
2medicines
0carried consumer input
0individual submissions
No submission for this condition has carried consumer input, including the one made since the comments facility came into use.
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| taliglucerase alfa Long-term enzyme replacement therapy for adult and paediatric patients with a confirmed diagnosis of Type 1 Gaucher disease. | Nov 2014 | Not recommended | none received |
| miglustat Type 1 Gaucher disease in patients who cannot be or have not been treated successfully with enzyme replacement therapy (ERT). | Mar 2008 | Recommended | no section in document |
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 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.