Phelan-mcdermid syndrome
Clinical trials for Phelan-mcdermid syndrome explained in plain language.
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Gene therapy trial hopes to tackle rare Autism-Linked condition
Disease control Recruiting nowThis early-stage trial tests a single dose of JAG201 gene therapy, given directly into the brain fluid, for people with SHANK3 haploinsufficiency (Phelan-McDermid syndrome), a rare genetic condition causing developmental delays and autism-like symptoms. The study includes childre…
Matched conditions: PHELAN-MCDERMID SYNDROME
Phase 1/2 • Sponsor: Jaguar Gene Therapy, LLC • Aim: Disease control
Last updated Sep 05, 2026 00:00 UTC
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Hope for rare genetic disorder: new drug trial targets core symptoms
Disease control Recruiting nowThis study tests an oral drug called NNZ-2591 in 160 children aged 3 to 12 with Phelan-McDermid syndrome, a rare genetic condition. The drug is compared to a placebo to see if it improves communication and daily function. The trial is double-blind, meaning neither the families no…
Matched conditions: PHELAN-MCDERMID SYNDROME
Phase 3 • Sponsor: Neuren Pharmaceuticals Limited • Aim: Disease control
Last updated Aug 27, 2026 00:00 UTC
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New drug shows promise for rare genetic disorder in kids
Disease control Recruiting nowThis phase 3 study is testing an oral drug called NNZ-2591 in 180 children aged 3 to 12 with Phelan-McDermid syndrome, a rare genetic condition that causes developmental delays and other challenges. The main goal is to see if the drug is safe and tolerable over the long term. Res…
Matched conditions: PHELAN-MCDERMID SYNDROME
Phase 3 • Sponsor: Neuren Pharmaceuticals Limited • Aim: Disease control
Last updated Aug 14, 2026 00:00 UTC