Alnylam RNAi Drug Reverses Progress of Rare Fatal Disease: Study
November 2, 2017
(Reuters) – More than half of patients with a rare, rapidly progressing fatal genetic disorder experienced significant improvement after being treated with an Alnylam Pharmaceutical Inc drug that uses gene silencing technology, according to results of a late stage study released on Thursday. The drug, patisiran, was being tested against the life-shortening condition called hereditary ATTR (hATTR) amyloidosis with polyneuropathy, which affects an estimated 50,000 people worldwide. It is being developed in partnership with Sanofi.