Disease index

Gaucher disease type 1

Metabolic · 2012–2015

4decisions
3medicines
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

MedicineMeetingOutcomeConsumer input
eliglustat Treatment of Gaucher disease type 1 (GD1) in adult patients aged ≥18 years with at least one GD1-related disease manifestation (skeletal disease,… Jul 2015 Not recommended none received
taliglucerase alfa Long-term enzyme replacement therapy for patients with a confirmed diagnosis of Gaucher disease. The submission sought listing for enzyme replacement therapy… Mar 2012 Not recommended unclear
velaglucerase alfa treatment of type 1 Gaucher disease in a patient who meets certain criteria, as per the Life Saving Drugs Program (LSDP) 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… Mar 2012 Deferred 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 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.