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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Gene therapy for sanfilippo a: does it last?
Disease control By invitation onlyThis study follows 41 children with Sanfilippo A (MPS IIIA) who previously received UX111 gene therapy in earlier trials. Researchers will monitor safety and how well the therapy controls the disease over time, using tests like the Bayley cognitive scale. No new gene therapy is g…
Phase 3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jul 19, 2026 00:00 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC