PITT HOPKINS SYNDROME

Clinical trials for PITT HOPKINS SYNDROME explained in plain language.

Never miss a new study

Get alerted when new PITT HOPKINS SYNDROME trials appear

Sign up with your email to follow new studies for PITT HOPKINS SYNDROME, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Trials to join now! 1 Not yet recruiting 1

Breakthrough gene therapy trial offers hope for rare genetic disorder

Disease control Recruiting now

This early-stage study is testing a new gene therapy called MZ-1866 for people with Pitt Hopkins Syndrome, a rare genetic disorder that affects brain development. Twelve children and adults will receive a single injection of the therapy directly into the fluid around the brain. R…

Matched conditions: PITT HOPKINS SYNDROME

Phase: PHASE1, PHASE2 • Sponsor: Mahzi Therapeutics • Aim: Disease control

Last updated Apr 03, 2026 14:43 UTC

PITT HOPKINS SYNDROME

Get alerted when new PITT HOPKINS SYNDROME trials appear

Breakthrough gene therapy trial offers hope for rare genetic disorder