Article
Cardiac responses in paediatric Pompe disease in the ADVANCE patient cohort.
Cardiology in the young - 1 Mar 2022
Byrne Barry J, Colan Steven D, Kishnani Priya S, Foster Meredith C, Sparks Susan E, Gibson James B, An Haack Kristina, Stockton David W, Peña Loren D M, Hahn Si Houn, Johnson Judith, Tanpaiboon Pranoot X, Leslie Nancy D, Kronn David, Hillman Richard E, Wang Raymond Y
Abstract excerpt
Pompe disease results from lysosomal acid α-glucosidase deficiency, which leads to cardiomyopathy in all infantile-onset and occasional late-onset patients. Cardiac assessment is important for its diagnosis and management. This article presents unpublished cardiac findings, concomitant medications, and cardiac efficacy and safety outcomes from the ADVANCE study; trajectories of patients with abnormal left...
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