Hurler-Scheie syndrome
MONDO:0011759Hurler-Scheie syndrome is the intermediate form of mucopolysaccharidosis type 1 (MPS1) between the two extremes Hurler syndrome and Scheie syndrome ; it is a rare lysosomal storage disease, characterized by skeletal deformities and a delay in motor development.
Also known as: Hurler-Scheie syndrome, MPS I H-S, MPS1H/S, MPSIH/S, mucopolysaccharidosis type 1H/S, mucopolysaccharidosis type IH/S, mucopolysaccharidosis, mps-I-s, Hurler–Scheie syndrome
5 clinical trials for this condition and its sub-types, 2 tagged with Hurler-Scheie syndrome itself.
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First human trial launches for Gene-Based MPS i treatment
Disease control Recruiting nowThis early-stage study tests whether ISP-001 is safe and tolerable in 11 people with a rare genetic disease called MPS I (Hurler-Scheie or Scheie types). Participants receive the experimental treatment and are monitored for side effects. The goal is to gather safety data for futu…
Phase 1 • Sponsor: Immusoft of CA, Inc. • Aim: Disease control
Last updated Jul 19, 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