New hope for rare genetic disorder: major trial tests daily pill for kids
Disease control
Recruiting now
This 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…
Phase: PHASE3 • Sponsor: Neuren Pharmaceuticals Limited • Aim: Disease control
Last updated Apr 22, 2026 15:58 UTC