Experimental drug tested for rare neurological disease
NCT ID NCT03759678
Summary
This study tested an experimental drug called N-Acetyl-L-Leucine for people with Ataxia-Telangiectasia (A-T), a rare genetic disorder that affects coordination and the immune system. The main goal was to see if the drug was safe and if it could help improve symptoms like walking, hand movements, and quality of life. All 17 participants received the drug for 6 weeks, followed by a 6-week period without it, so researchers could compare their condition with and without treatment.
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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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