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FDA approves first targeted RNA-based treatment for rare, fatal genetic disease

16:22 EDT 10 Aug 2018 | News-Medical.net

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.

Original Article: FDA approves first targeted RNA-based treatment for rare, fatal genetic disease

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