MUCOPOLYSACCHARIDOSIS III
Clinical trials for MUCOPOLYSACCHARIDOSIS III explained in plain language.
Never miss a new study
Get alerted when new MUCOPOLYSACCHARIDOSIS III trials appear
Sign up with your email to follow new studies for MUCOPOLYSACCHARIDOSIS III, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
Genom att skicka in godkänner du våra Användarvillkor
-
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: MUCOPOLYSACCHARIDOSIS III
Phase: PHASE2, PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 26, 2026 11:09 UTC
-
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: MUCOPOLYSACCHARIDOSIS III
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
-
French study aims to map rare MPS diseases in 1000 patients
Knowledge-focused Recruiting nowThis study is collecting health information from 1000 people in France with mucopolysaccharidosis (MPS), a group of rare genetic disorders. Researchers will look at medical records and test results to better understand how the diseases progress and how current treatments affect p…
Matched conditions: MUCOPOLYSACCHARIDOSIS III
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated May 26, 2026 10:59 UTC