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

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

Format: Article
Diterbitkan: Nature Portfolio 2022-08-01

Deskripsi

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.