Effects of enzyme replacement therapy on cardiac function in classic infantile Pompe disease.
Scheffers LE, Kok R, van den Berg LE, van den Hout JMP, Boersma E, van Capelle CI, Helbing WA, van der Ploeg AT, Koopman LP
International journal of cardiology, 2023 Mar 08
Abstract
Patients with classic infantile Pompe disease are born with a hypertrophic cardiomyopathy, which resolves after treatment with Enzyme replacement therapy (ERT). We aimed to assess potential deterioration of cardiac function over time using myocardial deformation analysis.