Recombinant human (alpha-glucosidase) from rabbit milk in Pompe patients

Pompe's disease is a fatal muscular disorder caused by lysosomal alpha-glucosidase deficiency. In an open-label study, four babies with characteristic cardiomyopathy were treated with recombinant human alpha-glucosidase (rhGAA) from rabbit milk at starting doses of 15 mg/kg or 20 mg/kg, and lat...

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Bibliographic Details
Published in:	The Lancet (British edition) 2000-07, Vol.356 (9227), p.397
Main Authors:	Hannerieke Van den Hout, Reuser, Arnold J J, Vulto, Arnold G, M Christa B Loonen
Format:	Article
Language:	eng
Subjects:	Disease Drug therapy Enzymes Muscular system
Online Access:	Get full text
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Summary:	Pompe's disease is a fatal muscular disorder caused by lysosomal alpha-glucosidase deficiency. In an open-label study, four babies with characteristic cardiomyopathy were treated with recombinant human alpha-glucosidase (rhGAA) from rabbit milk at starting doses of 15 mg/kg or 20 mg/kg, and later 40 mg/kg. The enzyme was generally well tolerated. Activity of alpha-glucosidase normalised in muscle. Tissue morphology and motor and cardiac function improved. The left-ventricular-mass index decreased significantly. We recommend early treatment. Long-term effects are being studied.
ISSN:	0140-6736 1474-547X