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 WilcoxPublished 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
- newly diagnosed
- ejection fraction
- chronic kidney disease
- peritoneal dialysis
- prognostic factors
- risk factors
- left ventricular
- heart failure
- replacement therapy
- emergency department
- skeletal muscle
- metabolic syndrome
- insulin resistance
- hypertrophic cardiomyopathy
- adverse drug
- patient reported