Mucopolysaccharidosis type 3A
MONDO:0009655A rare autosomal recessive lysosomal storage disease caused by deficiency of the enzyme heparan sulfate sulfatase. It is characterized by behavioral changes, sleep disturbances, mental developmental delays and seizures.
Also known as: MPS III A, MPS3A, MPSIIIA, Sanfilippo A, Sanfilippo syndrome a, Sanfilippo syndrome type A, heparan sulfamidase deficiency, mucopolysaccharidosis type 3A
10 clinical trials for this condition and its sub-types, 7 tagged with Mucopolysaccharidosis type 3A itself.
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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…
Phase 2/3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Aug 23, 2026 00:00 UTC
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MPS patients help design exercise program to boost mental health
Knowledge-focused Recruiting nowThis study aims to create a physical activity and sedentary behaviour program tailored for adults with mucopolysaccharidosis (MPS). Researchers will gather input from patients, doctors, nurses, and family members through interviews, focus groups, and workshops. The goal is to des…
Sponsor: Brunel University • Aim: Knowledge-focused
Last updated Aug 21, 2026 00:00 UTC