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.