Cardiac outcome in classic infantile Pompe disease after 13 years of treatment with recombinant human acid alpha-glucosidase

Carine I. van Capelle, Esther Poelman,Ingrid M. Frohn-Mulder, Laurens P. Koopman,Johanna M.P. van den Hout, Luc Régal,Bjorn Cools, Wim A. Helbing,Ans T. van der Ploeg

International Journal of Cardiology（2018）

引用 23|浏览28

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摘要

•ERT significantly reduced cardiac hypertrophy in classic infantile Pompe patients.•This significant improvement of LVMI is sustained over 14 years.•After start of treatment LVMI may initially increase, before starting to decline.•The risk for rhythm disturbances remains and warrants follow-up.

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关键词

Classic-infantile Pompe disease,Acid alpha-glucosidase,Enzyme replacement therapy (ERT),Hypertrophic cardiomyopathy,Tachyarrhythmia

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Cardiac outcome in classic infantile Pompe disease after 13 years of treatment with recombinant human acid alpha-glucosidase

Cardiac outcome in classic infantile Pompe disease after 13 years of treatment with recombinant human acid alpha-glucosidase