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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:
-
alpha-N-acetyl-D-glucosaminidase (alpha-N-acetylglucosaminidase)
:
2 studies in 35 results
: IBA
-
GALNS deficiency
:
1 study in 6 results
: IBA
-
genistin
:
1 study in 1 result
: IBA
-
Heparitin Sulfate (Heparan Sulfate)
:
45 results
: IBA
-
N-sulfoglucosamine sulfohydrolase (sulfamidase)
:
23 results
: IBA
-
Enzymes
:
15 results
: IBA
-
Glycosaminoglycans
:
6 results
: IBA
-
Acetylglucosaminidase (NAGase)
:
6 results
: IBA
-
Hexosaminidases (Hexosaminidase)
:
4 results
: IBA
-
heparan sulfate sulfatase
:
4 results
: IBA
Show All >>
Diseases Related to Mucopolysaccharidosis III
-
Mucopolysaccharidosis III (Sanfilippo Syndrome)
-
Mucopolysaccharidosis IV (Morquio Syndrome)
-
Mucopolysaccharidosis VII (Sly Syndrome)
-
Mucopolysaccharidoses
-
Lysosomal Storage Diseases (Lysosomal Storage Disease)
Show All >>
Key Therapies for Mucopolysaccharidosis III
Efficacy Chart >>
-
Transplantation (Transplant Recipients)
:
1 study in 2 results
-
Enzyme Replacement Therapy
:
1 study in 1 result
-
Investigational Therapies (Experimental Therapy)
:
1 result
-
Transplants (Transplant)
:
1 result
-
Hematopoietic Stem Cell Transplantation
:
1 result
Show All >>
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