As part of our investment in the development of effective treatments for myotonic dystrophy, MDF is helping develop what is called "benefit/risk" information for regulatory agencies reviewing potential therapies.
MDF hosted the Myotonic Dystrophy Patient-Centered Therapy Development Meeting in Washington, D.C. on September 17, 2015. The regulatory workshop featured speakers from academia, industry and the FDA.
MDF Chief Science Officer Sharon Hesterlee shares her initial thoughts on the newly-released FDA draft guidance for industry on drug development for rare diseases.
The 21st Century Cures Act (H.R. 6) is a rare bipartisan initiative launched by the House Energy and Commerce Committee. While there are many factors that drove this effort, fundamentally it was a recognition that despite significant medical progress, there are 10,000 known diseases or conditions, but we only have cures and treatments for 500 of them.