Science in Rare Diseases
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Gaucher Disease
Gaucher disease is a rare, progressive, inherited, genetic condition that causes a fatty substance, called glucosylceramide ( GL-1), to build up in various tissues including liver, spleen, and bone marrow. Explore this section to know more about Gaucher disease.
MPS-I
The mucopolysaccharidoses (MPS) family is a group of inherited diseases, each caused by accumulation of various glycosaminoglycans (GAGs) in the lysosomes. One of the important common type of MPS family - MPS I is caused by genetic changes in the IDUA gene. Explore relevant scientific content on MPS-I in this section.
