Mucopolysaccharidosis
MONDO:0019249A group of autosomal recessive or X-linked inherited lysosomal storage disorders affecting the metabolism of mucopolysaccharides, resulting in the accumulation of mucopolysaccharides in the body. Signs and symptoms include organomegaly, mental retardation, abnormal skeletal development, heart disorders, hearing loss, and central nervous system deficiencies.
Also known as: Mucopolysaccharidoses, mucopolysaccharidoses, mucopolysaccharidosis, MPS
61 clinical trials for this condition and its sub-types, 14 tagged with Mucopolysaccharidosis itself.
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Sub-types of Mucopolysaccharidosis
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Mucopolysaccharidosis type 2 24 trials
2 sub-types
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Mucopolysaccharidosis type 3 7 trials · 18 incl. sub-types
4 sub-types
- Mucopolysaccharidosis type 3A 7 trials
- Mucopolysaccharidosis type 3B 6 trials
- Mucopolysaccharidosis type 3C 2 trials
- Mucopolysaccharidosis type 3D 0 trials
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Mucopolysaccharidosis type 1 11 trials · 16 incl. sub-types
3 sub-types
- Hurler syndrome 6 trials
- Hurler-Scheie syndrome 2 trials
- Scheie syndrome 1 trial
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Mucopolysaccharidosis type 4 2 trials · 10 incl. sub-types
3 sub-types
- Mucopolysaccharidosis type 4A 8 trials
- Morquio syndrome C 0 trials
- Mucopolysaccharidosis type 4B 0 trials
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Mucopolysaccharidosis type 6 8 trials
2 sub-types
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Mucopolysaccharidosis type 7 8 trials
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Mucopolysaccharidosis type 9 1 trial
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Mucopolysaccharidosis, type 10 0 trials
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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