Patisiran

Patisiran, sold under the brand name Onpattro, is a medication for the treatment of polyneuropathy in people with hereditary transthyretin-mediated amyloidosis, a fatal rare disease that is estimated to affect 50,000 people worldwide.[1] It is the first small interfering RNA-based drug approved by the U.S. Food and Drug Administration (FDA) and the first drug approved by the FDA to treat this condition.[1] It is a gene silencing drug that interferes with the pr... [wikipedia]