Gaucher disease type 1
Rare disease · 2008–2015
7decisions
4medicines
0carried consumer input
0individual submissions
No submission for this condition has carried consumer input, including the 2 made since the comments facility came into use.
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| eliglustat Treatment of Gaucher disease type 1 in adult patients aged ≥18 years with at least one GD1-related disease manifestation including skeletal disease,… | Jul 2015 | Not recommended | none received |
| 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 |
| velaglucerase alfa Long-term enzyme replacement therapy for paediatric and adult patients with Type 1 Gaucher disease associated with anaemia, thrombocytopaenia, or… | Mar 2012 | Deferred | unclear |
| taliglucerase alfa Long-term enzyme replacement therapy for patients with a confirmed diagnosis of Gaucher disease, including Type 1 Gaucher disease associated with splenomegaly,… | Mar 2012 | Not recommended | unclear |
| velaglucerase alfa Long-term enzyme replacement therapy for paediatric and adult patients with Type 1 Gaucher disease associated with anaemia, thrombocytopenia, or… | Mar 2012 | Deferred | unclear |
| velaglucerase alfa Long-term enzyme replacement therapy for paediatric and adult patients with Type 1 Gaucher disease associated with at least one of the following clinical… | Nov 2011 | Deferred | no section in document |
| miglustat Type 1 Gaucher disease in patients who cannot be or have not been successfully treated 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 2 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.