Abstract
Mucopolysaccharidosis I (MPS I) is a rare disease with clinical manifestations in most systems. The disease is caused by a defect in the gene coding for the lysosomal enzyme α-L-iduronidase. This results in an inability of the lysosome to effect the stepwise degradation of glycosaminoglycans that alters the normal growth and homeostasis of tissues. The enzyme replacement therapy (ERT) with Laronidase is the only specific treatment for the disease than bone marrow transplant.
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