Author Archives: Marta Figueiredo PhD

Vutrisiran Reduces Neurologic Damage, Improves Physical Function, Trial Data Show

Vutrisiran, a second-generation RNA interference (RNAi) therapy candidate, safely and effectively reduces neurologic impairment and improves physical function and quality of life in adults with familial amyloid polyneuropathy (FAP). That’s according to top-line, nine-month data from the global HELIOS-A Phase 3 trial. Alnylam Pharmaceuticals, Vutrisiran’s developer, plans…

FDA Approves Alnylam’s Onpattro (Patisiran) as FAP Therapy

The U.S. Food and Drug Administration (FDA) has approved Alnylam Pharmaceuticals’ Onpattro (patisiran) as the first-ever treatment for patients in the U.S. with familial amyloid polyneuropathy (FAP). “Today’s historic approval marks the arrival of a first-of-its kind treatment option for a rare and devastating condition with limited treatment options,”…