Triple Therapy with Darbepoetin Alfa, Idebenone, and Riboflavin in Friedreich’s Ataxia: an Open-Label Trial

Triple Therapy with Darbepoetin Alfa, Idebenone, and Riboflavin in Friedreich’s Ataxia: an Open-Label Trial. Javier Arpa, Irene Sanz-Gallego, Francisco J. Rodríguez-de-Rivera, Francisco J. Domínguez-Melcón, Daniel Prefasi, Javier Oliva-Navarro, Mar Moreno-Yangüela, Samuel I. Pascual-Pascual. The Cerebellum April 2013 DOI 10.1007/s12311-013-0482-y



Long-term statistically nonsignificant improvement of LVMI and stability of the echocardiographic parameters could be considered. Triple therapy may slow disease progression of FRDA.

Progress in gene therapy for neurological disorders

Progress in gene therapy for neurological disorders. Michele Simonato, Jean Bennett, Nicholas M. Boulis, Maria G. Castro, David J. Fink, William F. Goins, Steven J. Gray, Pedro R. Lowenstein, Luk H. Vandenberghe, Thomas J. Wilson, John H. Wolfe & Joseph C. Glorioso; Nature Reviews Neurology , | doi:10.1038/nrneurol.2013.56

Standard medical and surgical practice has not proved effective in curing or treating these diseases, and appropriate pharmaceuticals do not exist or are insufficient to slow disease progression. Gene therapy is emerging as a powerful approach with potential to treat and even cure some of the most common diseases of the nervous system.

Erythropoietin receptor (EpoR) agonism to treat a wide range of diseases.

Erythropoietin receptor (EpoR) agonism to treat a wide range of diseases. Sanchis-Gomar F, Perez-Quilis C, Lippi G.; Mol Med. 2013 Apr 11. doi: 10.2119/molmed.2013.00025.

Keywords: Erythropoietin receptor (EpoR), pleitropic actions, heart and cardiovascular diseases, neurodegenerative disorders (Parkinson and Alzheimer), spinal cord injury, stroke, diabetic retinopathy, rare diseases (Friedreich ataxia) side effects, non-hematopoietic EpoR agonists drugs (asialoEpo, Cepo and ARA 290)