Assessment of plasma lyso-Gb3 for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease.

Daniel G BichetJohannes M AertsChristiane Auray-BlaisHiroki MaruyamaAtul B MehtaNina SkubanEva KrusinskaRaphael Schiffmann

Published in: Genetics in medicine : official journal of the American College of Medical Genetics (2020)

Although used as a pharmacodynamic biomarker in research and clinical studies, plasma lyso-Gb3 may not be a suitable biomarker for monitoring treatment response in migalastat-treated patients.

Keyphrases

newly diagnosed
end stage renal disease
ejection fraction
chronic kidney disease
left ventricular
smoking cessation