FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease

The U.S. Food and Drug Administration today approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients. This is the first FDA-approved treatment for patients with polyneuropathy caused by hATTR, a rare, debilitating and often fatal genetic disease characterized by the buildup of abnormal amyloid protein in peripheral nerves, the heart and other organs. http://www.worldpharmanews.com/fda/4510-fda-approves-first-of-its-kind-targeted-rna-based-therapy-to-treat-a-rare-disease