Condition: Ataxia Telangiectasia · Sponsor: Timothy Yu
This project aims to evaluate the safety and efficacy of precision genetic therapy for patients with Ataxia-telangiectasia (A-T), a rare neurodegenerative disease caused by mutations in the ATM gene. The investigators will conduct a clinical trial to study the safety and efficacy of intrathecal administration of atipeksen, a targeted genetic therapy that restores ATM gene function in A-T individuals bearing the recurrent ATM c.7865C\>T variant. The aim of this study is to delay or forestall progression of neurologic symptoms in A-T and improving quality of life. Success will provide an empirical foundation for advancing additional precision genetic therapies for A-T and other neurodegenerative conditions.
This description comes directly from the study's public registry record.
Arya Newington · 617-919-7499 · Arya.Newingham@childrens.harvard.edu
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| Boston Children's Hospital | Boston, Massachusetts, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT07215416