The investigational RNAi therapeutic vutrisiran (Alnylam Pharmaceuticals) met its primary and secondary endpoints in a phase 3 study of adults with hereditary transthyretin-mediated amyloidosis (hATTR ...

The US Food and Drug Administration (FDA) has approved vutrisiran (Amvuttra, Alnylam Pharmaceuticals) for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in ...

Please provide your email address to receive an email when new articles are posted on . Wainua has received regulatory approval for polyneuropathy of hereditary transthyretin-mediated amyloidosis.

WILMINGTON, Del.--(BUSINESS WIRE)--Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed ...

Credit: Getty Images. Eplontersen is an investigational ligand-conjugated antisense medicine designed to reduce the production of TTR protein to treat both hereditary and nonhereditary forms of ATTR.

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has granted ...

Hereditary transthyretin amyloidosis (hATTR) is a rare inherited disorder that gets worse over time. It often affects the nerves, heart, liver, and kidneys, but it can also affect other organs.

The outlook for hATTR-PN can vary depending on the genetic mutation involved and how the condition first presents. Newer treatments are helping improve outcomes. hATTR is a rare, hereditary disease. A ...

FDA Approves Alnylam’s Onpattro for Polyneuropathy of hATTR Amyloidosis August 10, 2018 The FDA today granted Alnylam Pharmaceuticals approval for a first-in-class small interfering ribonucleic acid ...

Please provide your email address to receive an email when new articles are posted on . The FDA approved patisiran infusion for the treatment of adults with polyneuropathy caused by hereditary ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has granted ...