Implications of Clinical Trials: A View of the Panorama

Alan Percy, MD and Walter Kaufmann, MD. The advances in basic and clinical research over the past two decades since the identification of mutations in the gene for most individuals with Rett syndrome (RTT) has been astonishing. Within the past few years, we have seen the number of clinical trials involving potential disease-modifying pharmaceutical agents … Read More

Having recently joined Rettsyndrome.org, I am humbled and grateful for the opportunity to serve this very special community. My 30 years as a health care administrator has taught me that medical solutions to even the simplest condition can be complicated and challenging. For example, there are about 75 drugs to treat something as “simple” as … Read More