The project is the result of an initiative of Spanish people affected by this rare disease. FARA, one of the main patients' associations in the United States now joins the endeavour
The gene therapy-based research project to tackle Friedreich's ataxia launched in November in labs at the Institute for Research in Biomedicine (IRB), in Barcelona, and the "Centro de Biolog-a Molecular Severo Ochoa" (CBMSO), in Madrid, has received 100,000 US$ per year for two years from the Friedreich's Ataxia Research Alliance (FARA). FARA is one of the main patients' organisations in the United States and since 1998 it has provided a rigorous and solid funding programme for research projects all over the world that aim to tackle Friedreich's ataxia, a rare and serious hereditary neurodegenerative disease for which only palliative treatments are currently available.
The peculiarity is that the idea of the project came from those affected by the disease, patients and relatives, who, in their endeavours to find a cure got in touch with basic research groups in order to start a long-term project. The GENEFA Platform, in close collaboration with FEDAES and the BabelFAmily, started fund-raising efforts in May 2013, and in November signed an agreement with IRB and CMBSO, headed by Joan Guinovart, director of the IRB, and Margarita Salas, president of the "Fundaci-n Severo Ochoa".
"The monthly subscriptions of members of the GENEFA Platform form the basis of the funding; however, the organisation has also held a wide range of fund-raising events and activities and has been supported by donations from companies, associations, and relatives and friends of those affected by Friedreich's ataxia. We are all working hard towards finding a cure and now the collaboration of FARA guarantees the funding required for this purpose," explains Juan Carlos Baiges, on behalf of the members of the Platform.