PHELAN-MCDERMID SYNDROME
Clinical trials for PHELAN-MCDERMID SYNDROME explained in plain language.
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First human trial tests One-Shot gene therapy for rare genetic disorder
Disease control Recruiting nowThis early-stage study is testing a single dose of an experimental gene therapy called JAG201 in children aged 2-9 with SHANK3 deficiency, which causes Phelan-McDermid Syndrome. The main goal is to check if the treatment is safe and tolerable when injected into the fluid around t…
Matched conditions: PHELAN-MCDERMID SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Jaguar Gene Therapy, LLC • Aim: Disease control
Last updated Apr 23, 2026 08:59 UTC
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New hope for rare genetic disorder: major trial tests daily pill for kids
Disease control Recruiting nowThis study is testing whether a daily oral medicine called NNZ-2591 can help children with Phelan-McDermid syndrome. It will compare the drug to a placebo in about 160 children aged 3 to 12. The main goals are to see if the medicine improves overall symptoms and communication ski…
Matched conditions: PHELAN-MCDERMID SYNDROME
Phase: PHASE3 • Sponsor: Neuren Pharmaceuticals Limited • Aim: Disease control
Last updated Apr 22, 2026 15:58 UTC