Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes.

Derralynn A HughesDaniel G BichetRoberto GiuglianiRobert J HopkinEva KrusinskaKathleen NichollsIacopo OlivottoUlla Feldt-RasmussenNorio SakaiNina SkubanGere Sunder-PlassmannRoser TorraWilliam R Wilcox

Published in: Journal of medical genetics (2022)

The overall incidence of FACEs for patients during long-term treatment with migalastat compared favourably with historic reports involving ERT. Lower baseline eGFR was a significant predictor of FACEs.

Keyphrases

end stage renal disease
small cell lung cancer
ejection fraction
chronic kidney disease
risk factors
prognostic factors
emergency department
heart failure
type diabetes
metabolic syndrome
adipose tissue
adverse drug