New hope for rare disease: expanded access to experimental drug for A-T patients

NCT ID NCT07380165

First seen Feb 02, 2026 · Last updated Apr 30, 2026 · Updated 10 times

Summary

This program provides an experimental drug, levacetylleucine, to people with Ataxia-Telangiectasia (A-T), a rare and serious genetic disease with no approved treatments. It is for patients who cannot join clinical trials and have no other good options. The goal is to offer potential relief from symptoms and control the disease, but it is not a cure.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

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Conditions

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