An open-label pilot study of recombinant granulocyte-colony stimulating factor in Friedreich’s ataxia

Abstract

Work in a mouse model of Friedreich’s ataxia has shown that administration of the cytokine granulocyte-colony stimulating factor (G-CSF) could have beneficial neuroprotective effects. Here the authors perform a pilot study of Lenograstim (recombinant G-CSF) in patients with Friedreich’s ataxia.