Mucopolysaccharidosis type 2, severe form
MONDO:0016315Mucopolysaccharidosis type 2 (MPS2), severe form (MPS2S), is associated with a massive accumulation of glycosaminoglycans and a wide variety of symptoms including a rapidly progressive cognitive decline; it is most often fatal in the second or third decade.
Also known as: Hunter syndrome type A, MPS2A, MPSIIA, iduronate 2-sulfatase deficiency type A, mucopolysaccharidosis type 2, severe form, mucopolysaccharidosis type 2A, mucopolysaccharidosis type II, severe form, mucopolysaccharidosis type IIA
4 clinical trials for this condition and its sub-types.
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Gene therapy breakthrough offers hope for boys with rare brain disease
Disease control OngoingThis study tests a new gene therapy for boys with Hunter syndrome, a genetic disorder that damages the brain and body. The therapy uses the child's own blood stem cells, modified to produce the missing enzyme, and aims to stop disease progression. Five boys aged 3 to 22 months wi…
Phase: PHASE1, PHASE2 • Sponsor: University of Manchester • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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New study monitors heart risks in rare genetic disease
Knowledge-focused OngoingThis study follows 30 people with mucopolysaccharidoses (MPS) over three years to see how their heart and arteries change. Researchers use neck ultrasounds and blood tests to measure artery thickness, stiffness, and signs of inflammation. The goal is to better understand cardiova…
Sponsor: Children's Hospital of Orange County • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:09 UTC