Gout drug shows promise for rare brain disorder in tiny trial
NCT ID NCT03776656
First seen Mar 09, 2026 · Last updated Jun 16, 2026 · Updated 17 times
Summary
This study tested the gout medicine allopurinol in 8 people with a rare genetic condition called ADSL deficiency, which causes autism, seizures, and developmental delays. Researchers measured changes in thinking, daily skills, and seizure control over 12 months. The goal was to see if allopurinol can improve symptoms by lowering harmful substances linked to the disorder.
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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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Department of Pediatry. Reference centre of Hereditary diseases of the metabolism of child and adult. Necker - Enfants malades Hospital
Paris, 75015, France
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LA PITIE-SALPETRIERE Hospital, AP-HP
Paris, 75013, France
Conditions
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