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Pompe disease, enzyme replacement therapy

Winkel, L. P., Van den Hout, J. M., Kamphoven, J. H. et al. Enzyme replacement therapy in late-onset Pompe s disease a three-year follow-up. Ann Neurol 55 495-502, 2004. [Pg.694]

Myozyme (alglucosidase alfa) (US) Approval 2006 Alglucosidase alfa enzyme replacement therapy Pompe disease... [Pg.948]

Figure 26.3 GSD II, Pompe s disease. GSD II (autosomal recessive) is caused by a deficiency of acid a-(l 4) glucosidase, a lysosomal enzyme. Glycogen accumulates, causing cardiomegaly after 2-3 months. The Uver and muscle are also affected, causing generalised muscle weakness. Enzyme replacement therapy has been successful in Pompe s disease especially by reversing the pathology in cardiac muscle. Figure 26.3 GSD II, Pompe s disease. GSD II (autosomal recessive) is caused by a deficiency of acid a-(l 4) glucosidase, a lysosomal enzyme. Glycogen accumulates, causing cardiomegaly after 2-3 months. The Uver and muscle are also affected, causing generalised muscle weakness. Enzyme replacement therapy has been successful in Pompe s disease especially by reversing the pathology in cardiac muscle.
See other pages where Pompe disease, enzyme replacement therapy is mentioned: [Pg.693]    [Pg.507]    [Pg.511]    [Pg.331]    [Pg.244]    [Pg.525]    [Pg.518]    [Pg.251]    [Pg.730]    [Pg.707]   


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