MUCOPOLYSACCHARIDOSIS III
Clinical trials for MUCOPOLYSACCHARIDOSIS III explained in plain language.
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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: MUCOPOLYSACCHARIDOSIS III
Phase: PHASE2, PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 13:06 UTC
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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: MUCOPOLYSACCHARIDOSIS III
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
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Massive french study aims to unlock secrets of rare MPS diseases
Knowledge-focused Recruiting nowThis observational study will follow up to 1,000 people in France with mucopolysaccharidosis (MPS), a group of rare genetic disorders. Researchers will collect medical data from patient records and ongoing checkups to map how the diseases progress and how current treatments affec…
Matched conditions: MUCOPOLYSACCHARIDOSIS III
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC