Mucopolysaccharidosis type 3B
MONDO:0009656A rare autosomal recessive lysosomal storage disease caused by deficiency of the enzyme N-acetyl-alpha-D-glucosaminidase. It is characterized by behavioral changes, sleep disturbances, and mental developmental delays.
Also known as: MPS III B, MPS3B, MPSIIIB, N-acetyl-alpha-glucosaminidase deficiency, Sanfilippo B, Sanfilippo syndrome B, Sanfilippo syndrome type B, mucopolysaccharidosis type 3B
9 clinical trials for this condition and its sub-types, 6 tagged with Mucopolysaccharidosis type 3B itself.
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Can a weekly brain infusion slow sanfilippo syndrome?
Disease control Expanded accessThis early-access program tests whether a weekly infusion of tralesinidase alfa directly into the brain's fluid spaces is safe and tolerable for children with Sanfilippo syndrome type B, a rare genetic disorder that causes severe neurological decline. The study enrolls about 10 c…
Sponsor: Spruce Biosciences • Aim: Disease control
Last updated Jul 31, 2026 00:00 UTC
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New hope for kids with rare sanfilippo syndrome? early trial launches
Disease control Recruiting nowThis early-phase study tests a new drug called JR-446 in 10 children with mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B), a rare genetic disease that affects the brain and body. The drug is given through an IV and aims to be safe and possibly help manage the conditi…
Phase 1/2 • Sponsor: JCR Pharmaceuticals Co., Ltd. • Aim: Disease control
Last updated Jun 26, 2026 12:37 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