A team from the The Scripps Research Institute (TSRI) was able to reactivate a gene, the metabolic silencing of which is responsible for Friedreich ataxia, a rare autosomal recessive neurodegenerative disorder that leaves affected persons crippled and vulnerable to scoliosis, diabetes, and heart disease. The research team, led by Joel Gottesfeld, PhD, a professor in the Department of Molecular Biology at TSRI in La Jolla, California, set about identifying and testing compounds that inhibited histone deacetylases in lymphocytes from persons with Friedreich ataxia. They hit upon one-BML-210-that reactivated the frataxin gene.