hereditary transthyretin amyloidosis
Rare disease · 2023–2024
2decisions
2medicines
2carried consumer input
69individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| vutrisiran Treatment of hereditary transthyretin mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, defined by Familial Amyloid… | Nov 2024 | Recommended with restriction | 12 individuals Leukaemia Foundation |
| patisiran Treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. | Dec 2023 | Recommended | 57 individuals |
Who spoke
Leukaemia Foundation
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.