Specific therapy
Enzyme Replacement therapy
Rationale
Since the basis for Pompe disease is the lack or shortage of well functioning α-glucosidase, replacement of this enzyme is critical to successful therapy to address the underlying cause. The purpose of MyozymeTM (alglucosidase alfa) therapy in Pompe disease is to provide exogenous enzyme for uptake into lysosomes and facilitate the breakdown of glycogen. This rationale of replacing the enzyme has proven to be true for other lysosomal storage disorders. See management of Gaucher disease, management of Fabry disease or management of MPS I.
Please see the full prescribing information in Dutch or in French (PDF) for MyozymeTM.
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