Onpattro (patisiran)

Onpattro (patisiran) is a medication used to treat hereditary transthyretin amyloidosis, a genetic condition where abnormal protein deposits harm organs like the nerves and heart. It works by delivering small pieces of genetic material called siRNA to the liver, which then reduces the production of the faulty protein called transthyretin. This decrease helps slow or prevent organ damage caused by the protein buildup. Administered as an intravenous infusion, Onpattro can improve symptoms and quality of life for patients with this rare disease by targeting the root cause of the protein accumulation.