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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Enzyme therapy shows promise for rare disease in china
Disease control CompletedThis study tested a drug called Aldurazyme (laronidase) in 12 Chinese people with mucopolysaccharidosis I (MPS I), a rare genetic disorder. Participants received weekly infusions for 26 weeks to see if the treatment is safe and reduces waste products in urine. The goal is to mana…
Phase: PHASE4 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Jun 26, 2026 18:08 UTC
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Massive MPS i registry sheds light on rare disease
Knowledge-focused CompletedThis registry tracked over 1,400 people with MPS I, a rare genetic disorder, to better understand how the disease progresses and how treatments like enzyme replacement therapy work over time. It did not test a new drug but collected real-world data to help doctors improve patient…
Sponsor: Genzyme, a Sanofi Company • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:08 UTC