NCBDDD’s Division of Human Development and Disability is pleased to announce a rebroadcast and live question and answer session of the CDC Public Health Grand Rounds: Where in health is disability?…Continue
DuchenneConnect was recently awarded close to $1 million dollars in funding from PCORI, the Patient-Centered Outcomes Research Institute!
DuchenneConnect will be one of 29 members of PCORnet, the National Patient-Centered Clinical Research Network. The goal of PCORnet is to create a large, highly representative, national network for conducting clinical research. This is especially…Continue
Last week, Pat Furlong and I were invited by the FDA to their meeting on Complex Issues in Developing Drugs and Biological Products for Rare Diseases. I was asked to participate on the Tolerating Risk and Uncertainty in Pediatric Clinical Trials Panel, in recognition of the work PPMD has done…Continue
Though clinical trials aren’t for everyone, each trial helps move us toward better treatments. What do you think about clinical trials? Share your thoughts, so PPMD can give the best advice to professionals running trials.
At the recent World Muscle…Continue
We all know it: rare diseases, especially progressive pediatric disorders like Duchenne, are different than common diseases. The FDA knows it too, but they don’t have a plan for how to treat them differently. They are working on different ways to weigh the benefits and risks of treatments for…Continue