CAMBRIDGE, Mass., April 28, 2025--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced that the Committee for ...

On May 19, BridgeBio Pharma, Inc. (NASDAQ:BBIO) announced promising results from its Phase 3 trial of Acoramidis, showing its ability to increase serum transthyretin (TTR) levels and significantly ...

Pfizer is taking a curtain call for Vyndaqel, the low-dose version of its blockbuster tafamidis franchise for the rare heart disease transthyretin amyloid cardiomyopathy (ATTR-CM). Pfizer will ...

Please provide your email address to receive an email when new articles are posted on . ALXN2220, a novel investigational depleter monoclonal antibody, was granted fast track designation by the FDA.

ATTR phenotypes vary by genotype as well as by gene penetrance, age, and geographic location. A new analysis examined data from untreated, symptomatic patients who had 1 of 4 variants: F64L, I68L, ...

When you have transthyretin amyloid cardiomyopathy (ATTR-CM), proteins called amyloids build up in your heart, stiffening the muscle and interfering with its ability to pump blood throughout the body.

Over time, transthyretin amyloidosis (ATTR) can damage the nerves, heart, and other organs. This may lead to complications like heart problems, nerve damage, and trouble with everyday bodily functions ...