Tag Archives: Familial Amyloidotic polyneuropathy

December, 2017

11 December
FDA Expands Orphan Drug Designation for Alnylam’s Patisiran to Treatment of Transthyretin-Mediated Amyloidosis

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, announced today the U.S. Food and Drug Administration (FDA) has granted a request to amend the orphan drug designation for patisiran to the treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis). This is an expansion to patisiran’s prior designation which was for …
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21 April
Alnylam’s RNAi Drug Shows Promise in Halting Neuropathy Progression in Patients with a Rare Genetic Disorder

Alnylam Pharmaceuticals’ lead RNAi therapy demonstrated positive results in a mid-stage study in patients with a rare genetic disorder. The company announced initial 12-month clinical data from its ongoing Phase II OLE study evaluating its patisiran for treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) in patients with familial amyloidotic polyneuropathy …
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