Hope for rare brain disorder: old drug tested for autism and epilepsy
NCT ID NCT03776656
Summary
This study tested whether a drug called allopurinol could help people with a rare genetic condition called adenylosuccinate lyase deficiency (ADSL). The condition causes intellectual disability, autism, and epilepsy. Eight children and adults took the drug for a year to see if it improved their daily living skills, communication, and behavior, and reduced seizures and harmful chemicals in their body.
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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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