Please watch this video of Josh and donate whatever you can to help him and the thousands of people worldwide with FA (Friedreich’s ataxia), a debilitating, life-shortening, degenerative neuro-muscular disorder. We thank you from the bottom of our hearts! All you have to do is click on the following link to…
To demonstrate safety and efficacy of allotopic human ND4 for treatment of a Leber’s hereditary optic neuropathy (LHON) mouse model harboring the G11778A mitochondrial mutation.
Federal agencies pursue world-renowned leaders when they seek expert advice. A recent search by the U.S. Food and Drug Administration for one such expert ended with a University of Florida College of Medicine pediatric researcher.
A biotechnology company has raised $30 million to advance gene therapy treatments developed at the University of Florida Powell Gene Therapy Center. The funding also will help UF Health researchers bring a novel form of gene therapy to patients with Pompe disease, a rare form of muscular dystrophy.
