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Erythropoietin in Friedreich ataxia: No effect on frataxin in a randomized controlled trial

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:

Read More: Erythropoietin in Friedreich ataxia: No effect on frataxin in a randomized controlled trial

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