ATAXIA TELANGIECTASIA

Clinical trials for ATAXIA TELANGIECTASIA explained in plain language.

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Not yet recruiting 1 Not yet finished but already full! 1 Completed 1 Terminated 2

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…

Matched conditions: ATAXIA TELANGIECTASIA

Phase: PHASE1, PHASE2 • Sponsor: Timothy Yu • Aim: Disease control

Last updated May 07, 2026 18:41 UTC

ATAXIA TELANGIECTASIA

Get alerted when new ATAXIA TELANGIECTASIA trials appear

New gene-targeting drug aims to halt rare childhood brain disease