Mucopolysaccharidosis type 1
MONDO:0001586The most common type of mucopolysaccharidosis. It is inherited in an autosomal recessive pattern. It comprises a group of lysosomal storage diseases which includes the most severe form (Hurler syndrome) and the mildest form (Scheie syndrome).
Also known as: Alpha-L-iduronidase deficiency, MPS1, MPSI, Mucopolysaccharidosis Type I, lipochondrodystrophy, mucopolysaccharidosis type 1, mucopolysaccharidosis type I, Hurler syndrome
19 clinical trials for this condition and its sub-types.
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Broader categories
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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: PHASE1 • Sponsor: Immusoft of CA, Inc. • Aim: Disease control
Last updated Jul 19, 2026 00:00 UTC
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New registry aims to unlock secrets of rare childhood diseases
Knowledge-focused Recruiting nowThis study collects information from up to 250 patients with lysosomal storage diseases (like certain forms of MPS, Pompe, Gaucher, and Wolman disease) to understand how these conditions develop and respond to treatments given before birth. Researchers will track symptoms, lab re…
Sponsor: University of California, San Francisco • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:05 UTC
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Massive french study aims to unlock secrets of rare MPS diseases
Knowledge-focused Recruiting nowThis observational study will follow up to 1,000 people in France with mucopolysaccharidosis (MPS), a group of rare genetic disorders. Researchers will collect medical data from patient records and ongoing checkups to map how the diseases progress and how current treatments affec…
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC
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New study aims to uncover heart risks in rare MPS diseases
Knowledge-focused Recruiting nowThis study looks at how heart and blood vessel problems develop in people with MPS I and IVA. Over 4 years, 240 participants will get yearly heart ultrasounds, blood tests, and urine tests. The goal is to find reliable markers that can track heart health and guide future treatmen…
Sponsor: Children's Hospital of Orange County • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:14 UTC