Pompe disease
Rare disease · 2021–2021
1decision
1medicine
1carried consumer input
25individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| avalglucosidase alfa Long-term enzyme replacement therapy for the treatment of patients with Pompe disease (infantile-onset, juvenile-onset, and adult-onset Pompe disease with… | Nov 2021 | Not recommended | 25 individuals Australian Pompe Association · Rare Voices Australia |
Who spoke
Australian Pompe Association · Rare Voices Australia
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: 1 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.