Research Fellowship: Visual dissection of GAA-mediated mechanisms of FRDA repression and identification of novel candidate factors involved in frataxin function

Principal Researchers: Dr Michele Lufino and Dr Richard Wade-Martins,
Department of Physiology, Anatomy and Genetics, University of Oxford.

Developing a Balanced Business Model for Gene Therapy

HUMAN GENE THERAPY 22:1–4 (June 2011),DOI: 10.1089/hum.2011.2504

"Research and outcomes like these give hope not only to patients, but to doctors who have spent years developing gene therapies for diseases that have not responded as well to traditional therapy.The promise shown by the results also create hope that gene therapy can finally emerge as avibrant segment, after years in which biopharma companies, physicians, insurers, and government regulators have struggled to develop a sustainable business model."

Full text pdf

Friedreich's Ataxia: Phenotypic variability: 4-case reports (two sets of siblings)

Published in Fisioterapia. 2010;32:190-4. - vol.32 núm 04
Gago Fernández, I.; Seco Calvo, J.

Keywords: clinical phenotype, Friedreich's Ataxia, activities of Daily Life, Health Quality of Life, Questionnaire of Health, multisystem involvement, phenotypic variability.