Pitt Hopkins Syndrome
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Sign up freeTop drugs studied for this disease
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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 …
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CLINICAL_TRIALS trial_result · 2025-05-18Clinical Trial Update: An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001) [Phase 2]
Open-label Phase 2 study of NNZ-2591 for Pitt Hopkins Syndrome completed; results have been posted.
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CLINICAL_TRIALS trial_result · 2024-08-26Clinical Trial Update: MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders [Phase 2]
A Phase 2 clinical trial evaluating MTT in children with Pitt Hopkins syndrome and gastrointestinal disorders has completed and posted resul…
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CLINICAL_TRIALS trial_result · 2024-03-20Clinical Trial Update: Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders [Phase 2]
Phase 2 clinical trial update for microbiota transfer therapy in Pitt Hopkins syndrome with gastrointestinal disorders; current status unkno…
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CLINICAL_TRIALS trial_result · 2021-12-21Clinical Trial Update: Safety & Efficacy of AlloRx SC® in PTHS Patients [Phase 1, Phase 2]
Clinical trial update on safety and efficacy of AlloRx SC® in Pitt Hopkins Syndrome across Phase 1 and Phase 2.