Shank3 Haploinsufficiency
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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 · 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…