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Clinical trials sponsored by Mahzi Therapeutics, explained in plain language.
This study tests a new gene therapy called MZ-1866 for children and adults with Pitt Hopkins Syndrome, a rare genetic condition that affects development and breathing. The therapy is given as a single injection into the fluid around the brain. The main goal is to see if it is saf…
Phase 1/2 • Sponsor: Mahzi Therapeutics • Aim: Disease control
Last updated Aug 02, 2026 00:00 UTC