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Patients and scientists join forces to tackle Friedreich's Ataxia
Date:11/14/2013

Barcelona, Thursday 14 November 2013.- The Spanish Federation of Ataxia (FEDAES)in representation of the GENEFA Platform for a Friedreich's Ataxia cure, the Babel Family association for biomedical research into Friedreich's Ataxia, the "Centro de Biologa Molecular Severo Ochoa" (CMBSO), and the Institute for Research in Biomedicine (IRB Barcelona) have signed an agreement through which these patients' associations will fund, by means of donations, a 3-year research project addressing Friedreich's Ataxia.

Friedreich's Ataxia is a rare degenerative disease of the nervous system that affects coordination, balance and movement. It is a monogenic disease, that is to say, it is caused by a defect in only one gene. Those affected by this disorder have inherited an altered frataxin gene from both parents. The project aims to develop molecular tools to transport a correct copy of the defective gene to all the cells of the body and particularly to a kind of neuron that undergoes degeneration and causes the disease. This approach seeks to restore the normal levels of frataxin and to stop the manifestation of the degenerative symptoms of the disease.

The GENEFA Platform heads the money-raising campaign to collect the 300,000 euros required to develop this gene therapy project. Juan Carlos Baiges, in representation of FEDAES/GENEFA and the Babel Family for this project, expresses his enthusiasm, "it is the first step towards achieving an effective treatment based on solid basic research knowledge", and adds, "we have a motivating project ahead that may lead us closer to a treatment."

Ernest Giralt, at IRB Barcelona, and Javier Daz-Nido, at CBMSO, scientific co-leaders of the project, stress that, "it is uncommon for basic researchers to have direct contact with patients and this project is fantastic because it reminds us that the solutions to diseases derive from basic research, the cornerstone of future applications."

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Contact: Sònia Armengou
armengou@irbbarcelona.org
34-934-037-255
Institute for Research in Biomedicine (IRB Barcelona)
Source:Eurekalert  

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