neuronal ceroid lipofuscinosis type 2
Rare disease · 2018–2018
1decision
1medicine
1carried consumer input
446individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| cerliponase alfa Treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a rare neurodegenerative genetic condition caused by deficiency of the enzyme TPP1. | Jul 2018 | Not recommended | 446 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: 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.