Nature Communications (Aug 2022)

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

  • Kevin C. Kemp,
  • Anastasia Georgievskaya,
  • Kelly Hares,
  • Juliana Redondo,
  • Steven Bailey,
  • Claire M. Rice,
  • Neil J. Scolding,
  • Chris Metcalfe,
  • Alastair Wilkins

DOI
https://doi.org/10.1038/s41467-022-31450-w
Journal volume & issue
Vol. 13, no. 1
pp. 1 – 8

Abstract

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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.