Safety and tolerability of carbamylated erythropoietin in Friedreich's ataxia.


BACKGROUND Erythropoietin (EPO) derivatives have been found to increase frataxin levels in Friedreich's ataxia (FRDA) in vitro. This multicenter, double-blind, placebo-controlled, phase II clinical trial aimed to evaluate the safety and tolerability of Lu AA24493 (carbamylated EPO; CEPO). METHODS Thirty-six ambulatory FRDA patients harboring >400 GAA… (More)
DOI: 10.1002/mds.25836