Los Angeles—An innovative new treatment that uses a natural process of gene silencing (RNA interference [RNAi]) has been shown to be highly effective in the treatment of patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy, a degenerative disorder that affects 50,000 people worldwide.
More than 90% of the haTTR patients who received an infusion of the investigational RNAi agent (patisiran; Sanofi and Alnylam Pharmaceuticals) every three weeks saw a halt in