PEDIATRICS Vol. 42 No. 4 October 1968, pp. 672-676
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Administration of a Mixture of Fungal Glucosidases to a Patient with Type II Glycogenosis (Pompe's Disease)

Ronald M. Lauer M.D.1, Thelma Mascarinas M.D.2, Antonio S. Racela M.D.2, Antoni M. Diehl M.D.2, and Barbara Illingworth Brown Ph.D.3

1 Department of Pediatrics, University Hospital, University of Iowa, Iowa City, Iowa 52240
2 Departments of Pediatrics and Pathology, University of Kansas Medical Center, Kansas City, Kansas 66103
3 Department of Biological Chemistry, Washington University School of Medicine, St. Louis, Missouri

A case of Type II glycogenosis (Pompe's disease) has been studied by histochemical, electron microscopic, and biochemical techniques. These studies have been made prior to and after the intramuscular administration for 1 week of a mixture of hydrolytic enzymes containing both agr-1,4- and agr-1,6-glucosidase activities.

Electron photomicrographs of the liver before enzyme administration showed glycogen to be located both within and outside of membrane-limited vacuoles. No change in this distribution could be detected in tissue removed by biopsy after enzyme administration. This impression was confirmed by the determination of glycogen content which was shown to be unchanged. Nevertheless, the liver was found by enzyme assay to contain the administered enzyme. Leucocytes isolated from blood taken 4 hours after the last enzyme injection were also shown to contain the parenterally administered glucosidases.

In skeletal muscle glycogen was present chiefly as extrasaccular deposits which were unchanged in appearance by enzyme administration. No glucosidase activity was demonstrable in the skeletal muscle after such a treatment.

Myocardium sectioned after autopsy had major deposits of glycogen in extrasaccular areas.




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