Drug repositioning screening identifies etravirine as a potential therapeutic for friedreich's ataxia

@article{Alfedi2019DrugRS,
  title={Drug repositioning screening identifies etravirine as a potential therapeutic for friedreich's ataxia},
  author={Giulia Alfedi and Riccardo Luffarelli and I. Cond{\`o} and G. Pedini and L. Mannucci and D. S. Massaro and M. Benini and N. Toschi and Giorgia Alaimo and Luca Panarello and L. Pacini and S. Fortuni and D. Serio and F. Malisan and R. Testi and A. Rufini},
  journal={Movement Disorders},
  year={2019},
  volume={34}
}
Friedreich's ataxia is an autosomal‐recessive cerebellar ataxia caused by mutation of the frataxin gene, resulting in decreased frataxin expression, mitochondrial dysfunction, and oxidative stress. Currently, no treatment is available for Friedreich's ataxia patients. Given that levels of residual frataxin critically affect disease severity, the main goal of a specific therapy for Friedreich's ataxia is to increase frataxin levels. 
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References

SHOWING 1-10 OF 68 REFERENCES
Progress in the treatment of Friedreich ataxia.
Friedreich's Ataxia: Autosomal Recessive Disease Caused by an Intronic GAA Triplet Repeat Expansion
Frataxin is reduced in Friedreich ataxia patients and is associated with mitochondrial membranes.
Pharmacological therapeutics in Friedreich ataxia: the present state
Friedreich's ataxia: Past, present and future
Translating HDAC inhibitors in Friedreich’s ataxia
Effect of diazoxide on Friedreich ataxia models
...
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3
4
5
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