Alnylam’s patisiran, the first ever FDA- and European Commission-approved RNAi therapeutic

Lipid nanoparticle structure On August 10, 2018, Alnylam Pharmaceuticals (Cambridge, MA) announced the first-ever FDA approval of an RNAi (RNA interference) drug. The drug is Alnylam’s patisiran, which is indicated for the treatment of polyneuropathy due to transthyretin-mediated amyloidosis (ATTR). ATTR is a rare inherited, debilitating, and often fatal disease caused by mutations [...]

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