hypophosphatasia
Rare disease · 2017–2018
2decisions
1medicine
2carried consumer input
61individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| asfotase alfa rch Treatment of juvenile-onset hypophosphatasia (HPP; onset between 6 months and 17 years of age). Asfotase alfa rch is used in combination with best supportive… | Mar 2018 | Not recommended | 55 individuals |
| asfotase alfa rch Treatment of patients with paediatric-onset hypophosphatasia (HPP), a rare genetic disorder of bone mineralisation. The submission addressed paediatric-onset… | Jul 2017 | Not recommended | 6 individuals |
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: 2 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.