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

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

Background The limited ability of enzyme replacement therapy (ERT) in removing globotriaosylceramide from cardiomyocytes is recognized for advanced Fabry disease cardiomyopathy (FDCM). Prehypertrophic FDCM is believed to be cured or stabilized by ERT. However, no pathologic confirmation is available...

Full description

Bibliographic Details
Main Authors: Andrea Frustaci, Romina Verardo, Nicola Galea, Maria Alfarano, Michele Magnocavallo, Livia Marchitelli, Luigi Sansone, Manuel Belli, Mario Cristina, Emanuela Frustaci, Matteo Antonio Russo, Cristina Chimenti
Format: Article
Language:English
Published: Wiley 2024-04-01
Series:Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease
Subjects:
enzyme replacement therapy
Fabry disease cardiomyopathy
globotrioasylceramide
mannose‐6‐phosphate receptors
Online Access:https://www.ahajournals.org/doi/10.1161/JAHA.123.032734

Internet

https://www.ahajournals.org/doi/10.1161/JAHA.123.032734

Similar Items