Long-Term Clinical-Pathologic Results of Enzyme Replacement Therapy in Prehypertrophic Fabry Disease Cardiomyopathy.

Andrea Frustaci Romina Verardo Nicola Galea Maria Alfarano Michele MagnocavalloLivia MarchitelliLuigi SansoneManuel Belli Mario CristinaEmanuela FrustaciMatteo Antonio Russo Cristina Chimenti

Published in: Journal of the American Heart Association (2024)

ERT stabilizes storage deposits and myocyte dimensions in 87% of patients with prehypertrophic FDCM. Globotriaosylceramide is never completely removed even after long-term treatment. Immune-mediated myocardial inflammation can overlap, limiting ERT activity.

Keyphrases

replacement therapy
smoking cessation
oxidative stress
heart failure
neoadjuvant chemotherapy
left ventricular
squamous cell carcinoma
locally advanced
radiation therapy
hypertrophic cardiomyopathy
combination therapy
rectal cancer