Records containing: Pitt Hopkins Syndrome
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2026-02-23Phase 1/2 First-in-Human Study to Evaluate the Safety, Tolerability, and Efficacy of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
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CLINICAL_TRIALS study record update · 2025-09-02An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome Using an "N of 1" Study Design
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CLINICAL_TRIALS study record update · 2025-05-29Coordination of Rare Diseases at Sanford
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CLINICAL_TRIALS study record update · 2025-05-18An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)
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CLINICAL_TRIALS study record update · 2025-04-04Early Check: A Collaborative Innovation to Facilitate Pre-Symptomatic Clinical Trials in Newborns
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CLINICAL_TRIALS study record update · 2024-08-26Microbiota Transfer Therapy for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
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CLINICAL_TRIALS study record update · 2024-03-20Microbiota Transfer Therapy for Children and Adults With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders: MTP-101 P
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CLINICAL_TRIALS study record update · 2021-12-21Randomized Double Blind Placebo Controlled Study of the Safety & Efficacy of Therapeutic Treatment With AlloRx Stem Cells® in Patients With Pitt Hopkins Syndrome (Phase 1/2 Study)