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Mucopolysaccharidoses Summary

Description: Group of lysosomal storage diseases each caused by an inherited deficiency of an enzyme involved in the degradation of glycosaminoglycans (mucopolysaccharides). The diseases are progressive and often display a wide spectrum of clinical severity within one enzyme deficiency.

Also Known As: Mucopolysaccharidosis

Networked: 1486 relevant articles (52 outcomes, 79 trials/studies) for this Disease

Key Drugs and Agents for Mucopolysaccharidoses

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. Glucuronidase : 9 outcomes 9 studies in 146 results : IBA
  2. Glycosaminoglycans : 6 outcomes 5 studies in 343 results : IBA
  3. Iduronidase (alpha-L-Iduronidase) : 3 outcomes 4 studies in 190 results : IBA
  4. idursulfase : 3 outcomes 4 studies in 20 results : FDA 2
  5. Heparitin Sulfate (Heparan Sulfate) : 3 outcomes 1 study in 115 results : IBA
  6. Enzymes : 2 outcomes 4 studies in 122 results : IBA
  7. dimethylmethylene blue : 2 outcomes in 6 results : IBA
  8. N-Acetylgalactosamine-4-Sulfatase (Arylsulfatase B) : 1 outcome 4 studies in 79 results : IBA
  9. Lysergic Acid Diethylamide (LSD) : 1 outcome 3 studies in 29 results : IBA
  10. GALNS deficiency : 1 outcome 2 studies in 59 results : IBA
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Diseases Related to Mucopolysaccharidoses

  1. Gaucher Disease (Gaucher's Disease)
  2. Mucopolysaccharidosis II (Hunter Syndrome)
  3. Cystic Fibrosis (Mucoviscidosis)
  4. Lysosomal Storage Diseases (Lysosomal Storage Disease)
  5. Mucopolysaccharidosis VI (Syndrome, Maroteaux-Lamy)
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Key Therapies for Mucopolysaccharidoses

Efficacy Chart >>
  1. Enzyme Replacement Therapy : 9 outcomes 18 studies in 138 results
  2. Bone Marrow Transplantation (Transplantation, Bone Marrow) : 5 outcomes 1 study in 53 results
  3. Intravenous Infusions : 2 outcomes 2 studies in 4 results
  4. Cell Transplantation : 2 outcomes 1 study in 11 results
  5. Hematopoietic Stem Cell Transplantation : 2 outcomes in 20 results
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