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Ambulatory electrocardiogram analysis in infants treated with recombinant human acid α-glucosidase enzyme replacement therapy for Pompe disease
Genetics in Medicine
Purpose: Infantile Pompe disease is caused by deficiency of lysosomal acid ␣-glucosidase. Trials with recombinant human acid ␣-glucosidase enzyme replacement therapy (ERT) show a decrease in left ventricular mass and improved function. We evaluated 24-hour ambulatory electrocardiograms (ECGs) at baseline and during ERT in patients with infantile Pompe disease. Methods: Thirty-two ambulatory ECGs were evaluated for 12 patients with infantile Pompe disease from 2003 to 2005. Patients had a mediandoi:10.1097/01.gim.0000217786.79173.a8 pmid:16702882 fatcat:m5xj55gpw5fdrmynbyisgwfrny