MUCOPOLYSACCHARIDOSIS III
Clinical trials for MUCOPOLYSACCHARIDOSIS III 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: MUCOPOLYSACCHARIDOSIS III
Phase: PHASE2, PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 17, 2026 08:23 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: MUCOPOLYSACCHARIDOSIS III
Phase: PHASE2, PHASE3 • Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center • Aim: Symptom relief
Last updated May 17, 2026 08:33 UTC
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French researchers launch largest-ever MPS patient registry to unlock disease secrets
Knowledge-focused Recruiting nowThis study will collect health information from 1,000 people in France with mucopolysaccharidosis (MPS), a group of rare genetic disorders. Researchers aim to better understand how the disease progresses and how it is currently managed with or without specific treatments. No new …
Matched conditions: MUCOPOLYSACCHARIDOSIS III
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Apr 29, 2026 15:03 UTC