Pitt Hopkins Syndrome
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CLINICAL_TRIALS research · 2026-02-23Clinical Trial Update: Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome [Phase 1, Phase 2]
This is an update on an early-phase clinical trial of MZ-1866, an AAV-9 gene therapy delivered by intracerebroventricular injection for Pitt…
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CLINICAL_TRIALS research · 2025-09-02Clinical Trial Update: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome [Phase 1]
A Phase 1 exploratory study is planned to evaluate the safety and potential efficacy of vorinostat in Pitt-Hopkins syndrome, with the trial …