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

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…

Matched conditions: ATAXIA TELANGIECTASIA

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

Last updated Apr 02, 2026 05:42 UTC

ATAXIA TELANGIECTASIA

Get alerted when new ATAXIA TELANGIECTASIA trials appear

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