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 Sep 21, 2026 13:01 UTC
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New hope for kids with sanfilippo syndrome b?
Disease control Recruiting nowThis 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 21, 2026 13:00 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