Mucopolysaccharidosis type 3
MONDO:0018937A lysosomal disease characterized by progressive neurocognitive decline, severe intellectual deterioration, loss of functional abilities, and premature death.
Also known as: MPS3, MPSIII, Mucopoly-saccharidosis type 3, Mucopolysaccharidosis Type III, Sanfilippo disease, Sanfilippo syndrome, heparan sulphate sulfatase deficiency, mucopolysaccharidosis type III
21 clinical trials for this condition and its sub-types, 7 tagged with Mucopolysaccharidosis type 3 itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Mucopolysaccharidosis type 3
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Mucopolysaccharidosis type 3A 7 trials
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Mucopolysaccharidosis type 3B 6 trials
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Mucopolysaccharidosis type 3C 2 trials
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Mucopolysaccharidosis type 3D 0 trials
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One-Time gene therapy aims to halt sanfilippo syndrome
Cure Not yet recruitingResearchers test a one-time gene therapy infusion in children and adults with Sanfilippo syndrome (MPS IIIB), a rare inherited disease that damages the brain and body. The therapy delivers a working copy of the NAGLU gene to help the body make an enzyme that is missing or faulty.…
Phase 1/2 • Sponsor: NeuroGT • Aim: Cure
Last updated Sep 16, 2026 00:00 UTC
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New hope for kids with sanfilippo syndrome b?
Disease control Not yet recruitingThis early-stage trial tests a new drug called JR-446 in 12 children under 6 with MPS IIIB, a rare genetic disorder that damages the brain and body. The main goal is to see if the drug is safe and tolerable. Researchers will also measure changes in certain substances in the body …
Phase 1/2 • Sponsor: JCR Pharmaceuticals Co., Ltd. • Aim: Disease control
Last updated Sep 10, 2026 00:00 UTC
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Hope for sanfilippo kids: Brain-Infused drug enters final testing
Disease control Not yet recruitingThis phase 3 trial tests a drug called tralesinidase alfa in 14 children aged 1-5 with Sanfilippo syndrome type B, a rare genetic disease that causes severe brain damage. The drug is given directly into the brain fluid to replace a missing enzyme. The goal is to see if it can slo…
Phase 3 • Sponsor: Spruce Biosciences • Aim: Disease control
Last updated Aug 01, 2026 00:00 UTC
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Smartphone videos could unlock secrets of rare brain disease
Knowledge-focused Not yet recruitingThis study tracks how Sanfilippo syndrome type C, a rare genetic disorder that affects the brain, progresses over time. Caregivers of children and young adults aged 1 to 25 will record videos of daily activities and answer questionnaires using a smartphone app every six months fo…
Sponsor: Phoenix Nest • Aim: Knowledge-focused
Last updated Jul 19, 2026 00:00 UTC
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New study aims to unmask hidden causes of childhood joint stiffness
Knowledge-focused Not yet recruitingThis study will look at 35 children with joint problems that are not caused by inflammation, such as stiffness or deformity. Researchers will use exams, lab tests, and imaging to find the true cause, which could be rare genetic conditions like mucopolysaccharidoses or osteogenesi…
Sponsor: Assiut University • Aim: Knowledge-focused
Last updated Jun 26, 2026 17:17 UTC