Alnylam Pharmaceuticals Alnylam develops RNA interference medicines that silence disease-causing genes, translating a once-experimental mechanism into six approved treatments for rare and prevalent diseases.
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Treats adults with transthyretin amyloidosis cardiomyopathy or hereditary transthyretin amyloidosis polyneuropathy through an approved RNAi medicine that reduces transthyretin production.
Treats primary hyperoxaluria type 1 in children and adults with an approved RNAi medicine that lowers oxalate production, urinary oxalate, and blood oxalate.
Prevents or reduces bleeding in hemophilia A or B through an approved antithrombin-lowering RNAi medicine discovered by Alnylam and licensed to Sanofi.
Provides an approved RNAi treatment for adults with hereditary transthyretin amyloidosis polyneuropathy, reducing production of the transthyretin protein underlying disease.
Treats acute hepatic porphyria in adults with an approved RNAi medicine that reduces production of toxic intermediates associated with porphyria attacks.
Lowers LDL cholesterol through an approved PCSK9-directed RNAi medicine discovered by Alnylam and licensed to Novartis for development and commercialization.
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