Keywords
mucopolysaccharidosis type I
How to Cite
Abstract
Lysosomal diseases are produced by the deficient activity of a lysosomal enzyme. With the advent of enzyme replacement therapy (ERT) for several lysosomal diseases, a specific therapeutic intervention was achieved. In the case of MPS type I, in 2003 both the FDA and the EMA approved the ERT with laronidasa (Aldurazyme ®-Genzyme Corporation). Given the protein nature of this enzyme, to ensure the success of this therapy it is necessary to strictly follow the patterns of storage, transportation, preparation and administration.References
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