Treating Hereditary TTR Amyloid Polyneuropathy with Patisiran

A five-year study on patisiran for hereditary transthyretin (TTR) amyloid polyneuropathy found that 65% of patients had stable or improved neuropathy, with better outcomes linked to early treatment. Despite efficacy, 19.4% of patients died. The study underscores the importance of early diagnosis and intervention in managing this progressive disease.