Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study
Hereditary transthyretin-mediated amyloidosis is a rare, inherited, progressive disease caused by mutations in the tranStHyretin (TTR) gene. The global OLE is ongoing with no new enrolment, and current findings are based on the interim analysis of the patients who had completed 12-month efficacy assessments as of the data cutoff. Continued long-term follow-up will be important for the overall assessment of safety and efficacy with patisirans
