What category of mutation—one that is associated with some degree of production of the alpha-galactosidase A (a-Gal A) enzyme—is amenable to the FDA-approved oral chaperone therapy, migalastat?

What category of mutation—one that is associated with some degree of production of the alpha-galactosidase A (a-Gal A) enzyme—is amenable to the FDA-approved oral chaperone therapy, migalastat?

What category of mutation—one that is associated with some degree of production of the alpha-galactosidase A (a-Gal A) enzyme—is amenable to the FDA-approved oral chaperone therapy, migalastat?


Created by

CMEducation Resources IQ&A Fabry Disease Diagnostic Intelligence Zone

Presenter

Robert Hopkin

Robert Hopkin

Associate Professor of Clinical Pediatrics
Cincinnati Children's Hospital Medical Center
Co-Director, 22Q-VCFS Center
University of Cincinnati College of Medicine
Cincinnati, OH