FDA Approves Targeted RNA-based Therapy to Treat Rare Disease

The U.S. Food and Drug Administration recently approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidoisis (hATTR) in adult patients. This rare debilitating and often fatal genetic disease is characterized by the buildup of abnormal amyloid protein in peripheral nerves, the heart and other organs. Onpattro is…

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Yale Researchers Make Breakthrough on Alzheimer’s Disease

Researchers at Yale University have tested a new method for directly measuring synaptic loss in individuals with Alzheimer’s disease. Published in the Journal of American Medical Association Neurology, the study was a collaborative effort between researchers at the Yale PET Center and the Yale Alzheimer’s Disease Research Unit. Alzheimer’s disease affects 5.7 million Americans, with…

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