Lysosomal storage disease with skeletal involvement
MONDO:0800088Also known as: dysostosis multiplex
64 clinical trials for this condition and its sub-types, 0 tagged with Lysosomal storage disease with skeletal involvement itself.
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Sub-types of Lysosomal storage disease with skeletal involvement
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Mucopolysaccharidosis type 2 25 trials
2 sub-types
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Mucopolysaccharidosis type 4A 8 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 3A 7 trials
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Hurler syndrome 6 trials
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Mucopolysaccharidosis type 3B 6 trials
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Alpha-mannosidosis 5 trials
3 sub-types
- Alpha-mannosidosis type 1 0 trials
- Alpha-mannosidosis, adult form 0 trials
- Alpha-mannosidosis, infantile form 0 trials
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Aspartylglucosaminuria 4 trials
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Mucosulfatidosis 4 trials
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GM1 gangliosidosis type 1 3 trials
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Hurler-Scheie syndrome 2 trials
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Fucosidosis 2 trials
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Galactosialidosis 2 trials
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Mucopolysaccharidosis type 3C 2 trials
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Scheie syndrome 1 trial
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GNPTG-mucolipidosis 0 trials
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Beta-mannosidosis 0 trials
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Mucolipidosis type II 0 trials
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Mucolipidosis type III, alpha/beta 0 trials
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Mucopolysaccharidosis type 3D 0 trials
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Mucopolysaccharidosis type 4B 0 trials
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Mucopolysaccharidosis-plus syndrome 0 trials
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Sialidosis type 2 0 trials
2 sub-types
- Congenital sialidosis type 2 0 trials
- Juvenile sialidosis type 2 0 trials
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Hope fades: trial of Tay-Sachs drug venglustat terminated early
Disease control Stopped earlyThis Phase 3 trial tested an oral drug called venglustat in 75 adults and children with late-onset Tay-Sachs or Sandhoff disease, rare genetic disorders that cause progressive nerve damage. The drug aimed to lower toxic fat buildup in the brain and slow disease worsening. However…
Phase 3 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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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