MDF Chief Science Officer Sharon Hesterlee shares her initial thoughts on the newly-released FDA draft guidance for industry on drug development for rare diseases.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.
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!
Dr. Sharon Hesterlee reports back on MDF's involvement with the Muscular Dystrophy Coordinating Committee.
For the second year in a row, MDF represented the DM community in Rare Disease Week activities on Capitol Hill and at the National Institutes of Health as part of the Rare Disease Legislative Advocates conference.