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11/06/2015 - 12:15pm

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.

10/10/2015 - 7:30am

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. 

09/10/2015 - 11:31am

MDF Chief Science Officer Sharon Hesterlee shares her initial thoughts on the newly-released FDA draft guidance for industry on drug development for rare diseases.

08/13/2015 - 12:38pm

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.

07/08/2015 - 4:51pm

On Friday, July 10, 2015, the 21st Century Cures Act (H.R. 6) passed the US House of Representatives with an overwhelming 344-77 majority!

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