Clinical Study Review: Metabolism in Friedreich’s ataxia (FA)
Recently I volunteered for a study with Dr. Shana McCormack at the University of Pennsylvania (UPenn) in collaboration with The Children’s Hospital of Philadelphia (CHOP). Dr. McCormack is an “Endocrinologist”, which means that she studies the how our bodies’ hormones control various and important bodily functions, such as: sleep, metabolism of food for energy generation, our mood, our growth and development, for example.
During my participation in this study, I thought about the difference between a “clinical study”, and a “clinical trial”. Dr. McCormack was not testing a new therapy (i.e., clinical trial), but was instead studying a feature of FA with the goal of enhancing our medical knowledge of the disease (i.e., clinical study). Armed with this knowledge, clinicians and researchers are empowered to develop better treatments for FA. The natural history study, for example, is a clinical study that has greatly improved our understanding of FA disease progression, and has positively influenced how Pharma companies design FA clinical trials. Just like when I volunteer for a clinical trial, I am confident that my participation in Dr. McCormack’s clinical study is getting us closer to treatments and a cure.Add a comment