Phelan-mcdermid Syndrome
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome [Phase 3]
The document reports on a Phase 3 clinical trial evaluating NNZ-2591 in pediatric participants with Phelan-McDermid Syndrome, with recruitme…
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CLINICAL_TRIALS research · 2026-02-09Clinical Trial Update: JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency [Phase 1, Phase 2]
Phase 1/2 clinical trial of JAG201 Gene Therapy for SHANK3 haploinsufficiency and Phelan-McDermid syndrome, currently recruiting.
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CLINICAL_TRIALS research · 2026-01-23Clinical Trial Update: Web Intervention for Parents of Youth With Genetic Syndromes (WINGS) [NA]
Rush University Medical Center is conducting a web-based intervention (WINGS) for parents of youths with several genetic syndromes, and the …
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CLINICAL_TRIALS research · 2025-08-13Clinical Trial Update: Multicenter Study of Patients With SHANK3 Mutations: Identification of Genes Modificators in Phelan-McDermid Syndrome (EUQ13) [NA]
A multicenter clinical trial investigating genetic modifiers in patients with SHANK3 mutations causing Phelan-McDermid syndrome; the study s…
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CLINICAL_TRIALS research · 2025-07-31Clinical Trial Update: Parent and Infant Inter(X)Action Intervention (PIXI) [NA]
Clinical trial update for the PIXI Inter(X)Action Intervention (NCT03836300), currently enrolling by invitation across Fragile X Syndrome, A…
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CLINICAL_TRIALS research · 2025-07-30Clinical Trial Update: RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS) [NA]
RB001 gene therapy is being studied in pediatric patients with SHANK3-related Phelan-McDermid Syndrome and the trial is currently recruiting…
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CLINICAL_TRIALS research · 2025-02-17Clinical Trial Update: Mapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome
This clinical trial update describes a study mapping genotype, phenotype, and natural history of Phelan-McDermid Syndrome, with related auti…