New gene-targeting drug aims to halt rare childhood brain disease
Disease control
Not yet recruiting
This early-stage trial tests a precision genetic medicine called atipeksen for people with Ataxia-Telangiectasia (A-T), a rare disease that damages the brain and nerves. The drug is given via spinal injection to restore a faulty gene. Up to 10 participants with a specific genetic…
Phase: PHASE1, PHASE2 • Sponsor: Timothy Yu • Aim: Disease control
Last updated May 07, 2026 18:41 UTC