Precision genetic therapy aims to slow rare brain disease
Disease control
Recruiting now
This early-stage trial tests a drug called atipeksen for people with Ataxia-Telangiectasia (A-T), a rare genetic disease that affects movement and coordination. The drug is designed to fix a specific genetic error in the ATM gene and is given via spinal injection. The study will …
Phase: PHASE1, PHASE2 • Sponsor: Timothy Yu • Aim: Disease control
Last updated Jun 27, 2026 12:38 UTC