- Early treatment with patisiran for hereditary transthyretin amyloidosis with polyneuropathy led to better clinical stability and survival.
- Investigaors enrolled 211 patients across 19 countries into a 5-year global open-label extension study, with 138 completing the global extension.
- Patients receiving patisiran exhibited a lower mortality rate.
- Multivariate analysis identified familial amyloid polyneuropathy stage and prior treatment as independent predictors of survival.
- Findings support the long-term safety and efficacy of RNA interference therapeutics in chronic disease management.
Early RNAi Therapeutic Treatment May Improve Survival in hATTR-PN
Conexiant
April 14, 2025