PITT HOPKINS SYNDROME
Clinical trials for PITT HOPKINS SYNDROME explained in plain language.
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New gene therapy hopes to change lives of children with rare brain disorder
Disease control Recruiting nowThis early-stage trial tests a new gene therapy called MZ-1866 for people with Pitt Hopkins Syndrome, a rare genetic condition that affects development. The study will include 12 children and adults who will receive a single injection of the therapy into the fluid around the brai…
Matched conditions: PITT HOPKINS SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Mahzi Therapeutics • Aim: Disease control
Last updated May 26, 2026 12:08 UTC
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Could a cancer drug help kids with rare pitt hopkins syndrome? small trial launches
Disease control Recruiting nowThis study tests whether vorinostat, a drug already used for cancer, is safe and helpful for people with Pitt Hopkins syndrome, a rare genetic condition that affects development. Five participants aged 3 to 21 will each receive placebo, a low dose, and a higher dose in a personal…
Matched conditions: PITT HOPKINS SYNDROME
Phase: PHASE1 • Sponsor: Unravel Biosciences, Inc. • Aim: Disease control
Last updated May 26, 2026 12:04 UTC