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Alnylam Gets Landmark FDA OK For First-Ever RNAi Drug

The FDA has just approved Alnylam’s patisiran (Onpattro), making it the first medicine specifically cleared to treat a rare and deadly disease called hereditary transthyretin amyloidosis (hATTR). The decision is a scientific milestone too: it marks the first-ever approval for a medicine that uses RNA interference (RNAi), a method cells can use to silence a gene before it makes a harmful protein. The FDA’s approval is specifically for the treatment of the peripheral nerve damage suffered by hATTR patients. The agency’s press release doesn’t mention the heart problems patients also often deal with.

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