BIOINTAXIS - A NEW COMPANY DEDICATED TO THE INVESTIGATION OF RARE DISEASES

In December 2018 the project successfully completed the pre-clinical phase where ataxia and the clinical signs were corrected with the transfer of frataxin protein to a mouse model with the disease, through the use of a vector based on adeno-associated virus (AAV), to the damaged neurons that are located mainly in the spinal cord and cerebellum. Our research is now being transferred in the design of a clinical trial for patients with Friedreich's Ataxia.