Natchez Hanson considers one of her proudest accomplishments walking across the stage to receive her college diploma earning a bachelors degree in math education. Hansen, 24, now a high school math teacher in Polk County, lives daily with the challenges of Friedreichs ataxia.
Friedreichs ataxia, a rare debilitating neuromuscular disease, typically strikes between the ages of 5 and 15, causing vision, balance, speech and cardiac problems and progressively robbing a young person of their energy, strength and ability to walk.
While physical therapy helps Hanson work on core strength and balance, there is no approved treatment for Friedreichs. Thats why she is so excited to be part of a USF-led national clinical trial of a drug that researchers, clinicians and patents hope will be the first to improve the symptoms of the life-shortening disease.
USF Health neurologist Dr. Theresa Zesiewicz, left, with patient Natchez Hanson, one of the participants in the USF-led national clinical trial for a potential Friedreichs ataxia drug.
I dont want anyone else to feel the way I did (when I was diagnosed) if they dont have to, Hanson said. I just cried because it was scary. A lot of people died being really young, and I was only 17. I had all these dreams I just wanted to be normal.
The double-blind, placebo-controlled trial, sponsored by Edison Pharmaceuticals, Inc., in collaboration with the Friedreichs Ataxia Research Alliance (FARA), is led by neurologist Dr. Theresa Zesiewicz, director of the USF Ataxia Research Center.
Researchers are primarily testing the effectiveness of the investigational drug EPI 743, a potent antioxidant, on vision, in patients with Friedreichs ataxia, many of whom experience varying degrees of visual changes. Secondarily, the study will evaluate neurological function.
Sixty patients with a genetically-confirmed Friedreichs ataxia diagnosis have been enrolled in the study, which involves Childrens Hospital of Philadelphia at the University of Pennsylvania and UCLA in Los Angeles, CA, as well as lead site USF.
When the study was announced at last years USF/FARA Scientific Symposium, the news was welcomed with cheers and tears by patients and their families who had come to learn about the latest advances in ataxia research.
The reaction and subsequent overwhelming international interest by prospective trial participants did not surprise Dr. Zesiewicz and others who care for those with Friedreichs ataxia.
See original here:
USF Leads National Clinical Trial Focusing on Friedreich's Ataxia