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 one-time gene therapy (UX111) for children with Sanfilippo syndrome type A, a rare genetic disorder that damages the brain. The treatment aims to reduce harmful substances in the brain and slow down the disease. About 36 children will take part in this advanced…
Matched conditions: SANFILIPPO SYNDROME
Phase: PHASE2, PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 17, 2026 06:50 UTC
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Could CBD ease agitation in rare childhood disease?
Symptom relief Recruiting nowThis study tests whether cannabidiol (CBD) can safely improve mood, behavior, and daily function 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 switch after a br…
Matched conditions: SANFILIPPO SYNDROME
Phase: PHASE2, PHASE3 • Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center • Aim: Symptom relief
Last updated May 17, 2026 07:23 UTC