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

08:51 EDT 10 Aug 2018 | FDA

FDA approves new drug for treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR). This is the first FDA-approved treatment for this rare, debilitating and often fatal genetic disease and the first FDA approval of a new class of drugs called small interfering ribonucleic acid (siRNA) treatment.

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

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