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BackgroundErythropoietin (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).
MethodsThirty‐six ambulatory FRDA patients harboring >400 GAA repeats were 2:1 randomly assigned to either...
Background:Friedreich ataxia is a rare disease caused by GAA‐trinucleotide‐repeat expansions in the frataxin gene, leading to marked reduction of qualitatively normal frataxin protein. Recently, human recombinant erythropoietin was reported to increase frataxin levels in patients with Friedreich ataxia.
Methods:We performed a 6‐month, randomized placebo‐controlled, double‐blind, dose‐response pilot...
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