In just two years, 21 MDF support groups have been launched throughout the U.S., Canada and Switzerland. Several more are poised to launch in early 2016 in Northern California, Dallas, South Miami, and Virginia. None of this would be possible without the dedicated facilitators who form the backbone of our community.
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MDF Chief Science Officer Sharon Hesterlee shares her initial thoughts on the newly-released FDA draft guidance for industry on drug development for rare diseases.