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  • Title: Improvement with ongoing Enzyme Replacement Therapy in advanced late-onset Pompe disease: a case study.
    Author: Case LE, Koeberl DD, Young SP, Bali D, DeArmey SM, Mackey J, Kishnani PS.
    Journal: Mol Genet Metab; 2008 Dec; 95(4):233-5. PubMed ID: 18930676.
    Abstract:
    Benefits of enzyme replacement therapy with Myozyme (alglucosidase alfa), anecdotally reported in late-onset Pompe disease, range from motor and pulmonary improvement in less severely affected patients, to stabilization with minimal improvement in those with advanced disease. We report a case of a 63-year-old patient with significant morbidity who made notable motor and pulmonary function gains after two years on therapy. Thus, improvements in those with advanced disease may be possible after long-term treatment.
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