Erythropoietin and Friedreich Ataxia: Time for a Reappraisal?

Boesch S and Indelicato E (2019); Front. Neurosci. 13:386. doi: 10.3389/fnins.2019.00386

Despite several clinical trials in the past, no treatment is available for the treatment of FRDA. Current lines of research focus on gene therapy, frataxin replacement strategies and on regulation of key metabolic checkpoints such as NrF2. Due to potential crosstalk with all these mechanisms, interventions on the EPO pathway still represent a valuable research field. The recent development of small EPO mimetics which maintain cytoprotective properties without erythropoietic action may open a new era in EPO research for the treatment of FRDA.



Erythropoietin and Friedreich Ataxia: Time for a Reappraisal?