SANFILIPPO SYNDROME
Clinical trials for SANFILIPPO SYNDROME explained in plain language.
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Gene therapy offers hope for kids with rare brain disease
Disease control Recruiting nowThis study tests a gene therapy called UX111 for children with Sanfilippo syndrome type A (MPS IIIA), a rare genetic disease that damages the brain. The treatment delivers a working gene to help the body break down harmful substances. The goal is to slow or stop the disease, but …
Matched conditions: SANFILIPPO SYNDROME
Phase: PHASE2, PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 26, 2026 10:38 UTC
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CBD shows promise for rare childhood brain disorder in new trial
Symptom relief Recruiting nowThis study tests whether cannabidiol (CBD) can safely improve behavior, mood, sleep, and other daily functions in people with Sanfilippo syndrome, a rare genetic disorder that affects the brain. About 35 participants aged 4 and older will receive either CBD or a placebo, then swi…
Matched conditions: SANFILIPPO SYNDROME
Phase: PHASE2, PHASE3 • Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center • Aim: Symptom relief
Last updated May 22, 2026 13:50 UTC