First-of-its-Kind genetic therapy aims to halt rare neurological disease
Disease control
Not yet recruiting
This early-stage study is testing a new genetic therapy called atipeksen for people with Ataxia-Telangiectasia (A-T), a rare and progressive neurological disease. The therapy is designed to correct a specific genetic error in the ATM gene, which causes A-T, with the goal of slowi…
Phase: PHASE1, PHASE2 • Sponsor: Timothy Yu • Aim: Disease control
Last updated Apr 02, 2026 07:26 UTC