SANFILIPPO SYNDROME
Clinical trials for SANFILIPPO SYNDROME explained in plain language.
Never miss a new study
Get alerted when new SANFILIPPO SYNDROME trials appear
Sign up with your email to follow new studies for SANFILIPPO SYNDROME, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
One-Time gene therapy aims to halt fatal brain disease in children
Disease control Recruiting nowThis trial tests a one-time gene therapy called UX111 for children with Sanfilippo A, a rare genetic disorder that causes severe brain damage. The therapy delivers a working copy of the missing gene to cells. Researchers will measure whether it reduces harmful substances in the b…
Matched conditions: SANFILIPPO SYNDROME
Phase: PHASE2, PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 13:06 UTC
-
CBD trial aims to ease sanfilippo syndrome symptoms
Symptom relief Recruiting nowThis study tests whether cannabidiol (CBD) can safely improve behavior, mood, sleep, and daily function in people with Sanfilippo syndrome, a rare genetic disorder. Thirty-five participants will receive either CBD or a placebo, then switch after a break. Caregivers will report on…
Matched conditions: SANFILIPPO SYNDROME
Phase: PHASE2, PHASE3 • Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center • Aim: Symptom relief
Last updated Jun 27, 2026 14:03 UTC