Holly Peay's Blog – October 2013 Archive (2)

Pushing the FDA to Accelerate Treatments

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 rare disease versus treatments for treat common…


Added by Holly Peay on October 10, 2013 at 9:30am — No Comments

Clinical Trial Expectations

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 improve the clinical trial process. Highlighting the…


Added by Holly Peay on October 7, 2013 at 10:58am — No Comments

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