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
8 clinical trials for this condition and its sub-types.
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Can a weekly brain infusion slow sanfilippo syndrome?
Disease control AVAILABLEThis 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: PHASE1, PHASE2 • Sponsor: JCR Pharmaceuticals Co., Ltd. • Aim: Disease control
Last updated Jun 26, 2026 12:37 UTC