First-of-its-Kind genetic therapy aims to halt rare neurological disease

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 slowing or stopping the worsening of symptoms. The study will enroll 10 participants with a particular ATM gene mutation to first check safety and see if the treatment, given via spinal injection, can help stabilize neurological function and improve quality of life.