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Alglucosidase alfa enzyme replacement therapy as a therapeutic approach for glycogen storage disease type III.
(Mol Genet Metab, 2013-02)
We investigated the feasibility of using recombinant human acid-α glucosidase (rhGAA, Alglucosidase alfa), an FDA approved therapy for Pompe disease, as a treatment approach for glycogen storage disease type III (GSD III). ...