The study involves adults diagnosed with Phelan-McDermid Syndrome, a rare genetic condition that can cause developmental delays, speech difficulties, and behavioral challenges. Participants will receive the oral medication vafidemstat, supplied in capsule form.
The purpose of the study is to evaluate the safety and tolerability of the medication in this population. Participants will take the capsules daily for a set period, attending regular visits where doctors will check vital signs, perform physical examinations, and record any side effects. Blood tests and a heart rhythm test called an electrocardiogram (ECG) will also be performed to monitor overall health.
In addition to safety checks, the study will use questionnaires such as the Aberrant Behaviour Checklist and the Clinical Global Impression of Severity to track changes in irritability, aggression, and other behaviors. Any thoughts of self‑harm will be assessed with the Columbia‑Suicide Severity Rating Scale (C-SSRS), a questionnaire that helps identify suicidal thoughts. All information collected will be used to determine whether the medication is safe and well‑tolerated for adults with the condition.



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