Introduction and Overview

Aldurazyme® (laronidase) is a form of recombinant human a-L-iduronidase (rhIDU) developed as an enzyme replacement therapy (ERT) for the rare recessive genetic disorder, mucopolysaccharidosis I (MPS I) [1]. The a-L-iduronidase enzyme (EC is normally located and active in the lysosome, where it participates in the sequential enzymatic degradation process of certain glycosaminoglycans (GAGs). Patients with MPS I are genetically deficient in a-L-iduronidase and accumulate undegraded GAG chains within lysosomes, leading to a profound and pervasive lysosomal storage disorder (Figure 12.1).

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