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

Locations

  • Department of Pediatry. Reference centre of Hereditary diseases of the metabolism of child and adult. Necker - Enfants malades Hospital

    Paris, 75015, France

  • LA PITIE-SALPETRIERE Hospital, AP-HP

    Paris, 75013, France

Conditions

Explore the condition pages connected to this study.