late-onset Pompe disease
Rare disease · 2009–2025
7decisions
2medicines
2carried consumer input
16individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| cipaglucosidase alfa with miglustat Treatment of adults with late-onset Pompe disease (LOPD), a rare autosomal recessive glycogen storage disorder caused by GAA gene mutations leading to… | Mar 2025 | Not recommended | 16 individuals |
| alglucosidase alfa Treatment of late-onset Pompe disease in patients with confirmed diagnosis based on enzymology or mutational analysis and clinical features of juvenile or… | Mar 2013 | Recommended | unclear |
| alglucosidase alfa treatment of late-onset Pompe disease | Nov 2012 | Recommended | no section in document |
| alglucosidase alfa Late-onset Pompe disease (acid alfa-glucosidase deficiency). Treatment aims to slow progressive muscle weakness and extend survival in patients with this rare… | Nov 2012 | Recommended | no section in document |
| alglucosidase alfa treatment of late onset Pompe disease with a documented deficiency of acid alpha-glucosidase enzyme activity in muscle tissues, confirmed by laboratory tests… | Nov 2010 | Not recommended | input received |
| alglucosidase alfa treatment of late-onset Pompe disease with a documented deficiency of acid alpha-glucosidase enzyme activity | Mar 2009 | Not recommended | no section in document |
| alglucosidase alfa Treatment of late-onset Pompe disease (acid alfa-glucosidase deficiency) in patients with significant deterioration in lung function (FVC <80% predicted) or… | Nov 2009 | 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: 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.