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Mucopolysaccharidosis III (Sanfilippo Syndrome) Summary

Description: Mucopolysaccharidosis characterized by heparitin sulfate in the urine, progressive mental retardation, mild dwarfism, and other skeletal disorders. There are four clinically indistinguishable but biochemically distinct forms, each due to a deficiency of a different enzyme.

Also Known As: Sanfilippo Syndrome; MPS III A; MPS III B; MPS III C; MPS III D Show All >>

Networked: 142 relevant articles (0 outcomes, 5 trials/studies) for this Disease

Key Drugs and Agents for Mucopolysaccharidosis III

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. alpha-N-acetyl-D-glucosaminidase (alpha-N-acetylglucosaminidase) : 2 studies in 35 results : IBA
  2. GALNS deficiency : 1 study in 6 results : IBA
  3. genistin : 1 study in 1 result : IBA
  4. Heparitin Sulfate (Heparan Sulfate) : 45 results : IBA
  5. N-sulfoglucosamine sulfohydrolase (sulfamidase) : 23 results : IBA
  6. Enzymes : 15 results : IBA
  7. Glycosaminoglycans : 6 results : IBA
  8. Acetylglucosaminidase (NAGase) : 6 results : IBA
  9. Hexosaminidases (Hexosaminidase) : 4 results : IBA
  10. heparan sulfate sulfatase : 4 results : IBA
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Diseases Related to Mucopolysaccharidosis III

  1. Mucopolysaccharidosis III (Sanfilippo Syndrome)
  2. Mucopolysaccharidosis IV (Morquio Syndrome)
  3. Mucopolysaccharidosis VII (Sly Syndrome)
  4. Mucopolysaccharidoses
  5. Lysosomal Storage Diseases (Lysosomal Storage Disease)
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Key Therapies for Mucopolysaccharidosis III

Efficacy Chart >>
  1. Transplantation (Transplant Recipients) : 1 study in 2 results
  2. Enzyme Replacement Therapy : 1 study in 1 result
  3. Investigational Therapies (Experimental Therapy) : 1 result
  4. Transplants (Transplant) : 1 result
  5. Hematopoietic Stem Cell Transplantation : 1 result
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