Advocacy

Community Perspectives on Future Drug Benefits and Risks

As part of our investment in the development of effective treatments for myotonic dystrophy, Myotonic is helping develop what is called "benefit/risk" information for regulatory agencies reviewing potential therapies.

Experts Participate in DM Regulatory Workshop

Myotonic 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. 

Congress Acts to Spur Research, Drug Development

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.

What is Orphan Drug Designation?

FDA's Orphan Drug Program is designed to encourage pharmaceutical companies to develop drugs for rare or 'orphan' diseases that might otherwise not be appealing to industry.

MD-CARE Act Signed Into Law!

On Friday, September 26, 2014, the MD-CARE Amendments were signed into law by President Obama!

© Myotonic Dystrophy Foundation. All rights reserved.