New drug aims to stop rare genetic disease before it strikes
Prevention
Recruiting now
This phase 3 trial tests whether acoramidis can prevent or delay transthyretin amyloidosis (ATTR) in 587 adults who carry a faulty gene but have no symptoms yet. ATTR causes sticky plaques to build up in the heart and nerves, leading to heart failure and nerve damage. Acoramidis …
Phase: PHASE3 • Sponsor: Eidos Therapeutics, a BridgeBio company • Aim: Prevention
Last updated Jul 01, 2026 00:00 UTC