Experimental drug shows promise for rare childhood movement disorder
NCT ID NCT03759678
First seen Mar 28, 2026 · Last updated May 15, 2026 · Updated 7 times
Summary
This study tested a drug called N-Acetyl-L-Leucine in 17 people with Ataxia-Telangiectasia (A-T), a rare genetic disease that causes severe movement problems. The goal was to see if the drug could safely improve symptoms like coordination and walking. The study was stopped early, but researchers looked at changes in movement ability over 6 weeks of treatment compared to a period without treatment.
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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.
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
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Locations
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Hospital Universitario La Paz
Madrid, Spain, Spain
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Royal Papworth Hospital NHS Foundation Trust
Cambridge, Cambridgeshire, CB2 0AY, United Kingdom
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University of California - Los Angeles
Los Angeles, California, 90095, United States
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University of Giessen
Giessen, Germany
Conditions
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