Website and Blog for the Sam McMahon Trust

The Repligen Corporation announced today that it has lodged an application with the Food and Drug Administration (FDA) for a Phase 1 study of RG2833, a selective histone deacetylase 3 (HDAC-3) inhibitor. Pending FDA approval this will be this first step in the clinical development of RG2833 as a potential treatment for FA.

Repligen state that ‘preclinical studies in animal models and patients’ cells have shown that RG2833 crosses the blood brain barrier, activates the defective frataxin gene and increases the production of frataxin.’

To read the full press release you can go the FARA website here and go to News on the right hand side of the page.

Check out this article in our Useful Information page, that was published in the January 2010 issue of  Neurology Now. This is a publication of the American Academy of Neurologists. Although it is not a comprehensive review of all of the research that is going on in the world of FA, it does acknowledge the promising work that is being done by Dr Joel Gottesfeld in the field of HDAC inhibitors.