Could a cancer drug ease social struggles in rare genetic disorder?
NCT ID NCT07218575
Summary
This study is testing if the drug everolimus can safely improve social skills in people with PTEN Hamartoma Tumor Syndrome (PHTS), a genetic condition often linked to autism-like social difficulties. For the first six months, half of the 60 participants will take everolimus pills and half will take placebo pills, with everyone receiving the drug for the final six months. Researchers will measure changes in social abilities using questionnaires filled out by family members or caregivers.
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