Gene therapy trial hopes to tame rare brain disorder
Disease control
Recruiting now
This early-stage trial tests a one-time gene therapy called MZ-1866 for children and adults with Pitt Hopkins Syndrome, a rare genetic condition affecting development. The study will enroll 12 participants to check if the treatment is safe and tolerable. Researchers will monitor …
Phase: PHASE1, PHASE2 • Sponsor: Mahzi Therapeutics • Aim: Disease control
Last updated Apr 30, 2026 15:51 UTC