Alnylam's lead product, Onpattro (patisiran), is an RNAi therapeutic approved for the treatment of hereditary transthyretin amyloidosis (hATTR), a rare and debilitating genetic disorder. Onpattro has demonstrated significant clinical benefits in patients with hATTR, including improvements in quality of life, ambulation, and cardiac function. Alnylam is also developing a pipeline of promising new RNAi therapeutics for a variety of other diseases, including liver diseases, cardiovascular diseases, and central nervous system (CNS) disorders.
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