New preclinical findings from extensive cell and animal studies suggest that a drug already used for a rare kidney disease could benefit patients with some mitochondrial disorders—complex conditions with severe energy deficiency for which no proven effective treatments exist. Future clinical research is needed to explore whether the drug, cysteamine bitartrate, will meaningfully benefit patients.
Super Bowl Sunday is right around the corner, and our biweekly installment of In the News is the perfect playing field for us to showcase the wide range of research projects that Children's Hospital of Philadelphia experts tackle. Huddle up!
The Wilson Lab studies the inherited neuro- and cardio-degenerative mitochondrial disorder, Friedreich ataxia (FA). The lab also constructed the first random shRNA-expressing libraries and is using these libraries for drug discovery in a variety of disorders.
