Mucopolysaccharidosis type 2
MONDO:0010674A lysosomal storage disease leading to a massive accumulation of glycosaminoglycans and a wide variety of symptoms including distinctive coarse facial features, short stature, cardio-respiratory involvement and skeletal abnormalities. It manifests as a continuum varying from a severe to an attenuated form without neuronal involvement.
Also known as: Hunter syndrome, Hunter's syndrome, I2S deficiency, IDS deficiency, MPS 2, MPS II, MPS with skin involvement, MPS2
28 clinical trials for this condition and its sub-types, 25 tagged with Mucopolysaccharidosis type 2 itself.
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Sub-types of Mucopolysaccharidosis type 2
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Home infusions may help patients stick to treatment
Knowledge-focused Stopped earlyThis study looks at whether people with Fabry, Gaucher, or Hunter disease are more likely to continue their IV treatment when it's given at home versus at a hospital. Researchers will review existing data from 222 patients in Mexico. No new treatments are given; the goal is to un…
Sponsor: Takeda • Aim: Knowledge-focused
Last updated Sep 13, 2026 00:00 UTC
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Gene Editing's lasting impact: a 10-Year safety watch
Knowledge-focused Stopped earlyThis study checks on people who previously received gene editing for hemophilia B or mucopolysaccharidosis (MPS) I or II. No new treatment is given; instead, participants are monitored for up to 10 years to see if any new health problems or worsening of existing conditions appear…
Sponsor: Sangamo Therapeutics • Aim: Knowledge-focused
Last updated Aug 15, 2026 00:00 UTC