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
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
I was privileged to share some of the data PPMD has collected in our Clinical Trial Expectations study at the World Muscle Society poster session. Many clinicians and industry and academic scientists came to the poster to learn about our community thoughts and preferences, and were quite interested in how they might…Continue
Earlier this week, PPMD hosted a webinar to discuss our FDA “aggressive collaboration” approach and the results from our recent benefit/risk survey. We conducted a rigorous survey of more than 100 parents whose children have Duchenne, gathering invaluable perspectives as to treatment priorities and risk threshold based on realistic but hypothetical treatments – information that PPMD is encouraging regulators to use to…Continue