Records containing: Phelan-McDermid 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 · 2026-04-13A Phase 3 Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of Orally Administered NNZ-2591 Compared With Placebo in Pediatric Participants With Phelan-McDermid Syndrome
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CLINICAL_TRIALS study record update · 2026-02-09A Phase 1/2, Multicenter, Open-Label, Dose-Escalation, Safety, Tolerability, and Clinical Activity Study of a Single Dose of JAG201 Gene Therapy Delivered Via Intracerebroventricular Administration in Participants With SHANK3 Haploinsufficiency
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CLINICAL_TRIALS study record update · 2025-07-31Piloting an Early Intervention Program for Infants With Rare Neurogenetic Disorders
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CLINICAL_TRIALS study record update · 2025-07-30An Open-label, Single Arm, Dose-Escalation Clinical Study to Evaluating the Safety, Tolerability and Preliminary Efficacy of a Single Intracerebroventricular Injection of RB001 for the Treatment of SHANK3-related Phelan McDermid Syndrome.
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CLINICAL_TRIALS study record update · 2025-05-30An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)
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CLINICAL_TRIALS study record update · 2025-04-20Electrophysiological Markers for Interventions in Phelan-McDermid Syndrome and Idiopathic Autism
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CLINICAL_TRIALS study record update · 2025-02-18Mapping the Phenotype in Adults With Phelan-McDermid Syndrome
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CLINICAL_TRIALS study record update · 2025-02-17Mapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome
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CLINICAL_TRIALS study record update · 2022-10-10Effectiveness of Recombinant Human Growth Hormone Therapy for Children With Phelan-McDermid Syndrome: An Open-label, Cross-over, Preliminary Study
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CLINICAL_TRIALS study record update · 2022-05-12A Double-Blind Placebo-Controlled Crossover Trial of Insulin-Like Growth Factor-1 (IGF-1) in Children and Adolescents With 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)
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CLINICAL_TRIALS study record update · 2021-12-06Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome
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CLINICAL_TRIALS study record update · 2021-08-05Mitochondrial Dysfunction in Phelan-McDermid Syndrome: Explaining Clinical Variation and Providing a Path Towards Treatment
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CLINICAL_TRIALS study record update · 2021-06-22An Open-label Study to Investigate the Safety, Tolerability and Efficacy of a Single 6-hour Intravenous Infusion of AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy
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CLINICAL_TRIALS study record update · 2020-03-18Randomized, Placebo-controlled, Cross-over, Double-blind Study of a Metabolic Support Therapy With Q10 Ubiquinol and a Multivitamin B and E Complex in Two Cohorts of Patients With Idiopathic and Syndromic Autism (Phelan-McDermid Syndrome)