In July, the U.S. House of Representatives overwhelmingly passed bipartisan legislation called the 21st Century Cures Act which seeks to accelerate the discovery, development, and delivery of life saving and life improving therapies. This represents an important milestone for individuals living with myotonic dystrophy. This year, MDF has been actively engaged in this effort and is now urging Senators to swiftly act on their version of the bill. Additionally, MDF is urging inclusion of the following House provisions in the Senate bill:
- Increased federal funding for the National Institutes of Health (NIH) and the Food and Drug Administration (FDA);
- A requirement that the FDA consider patient perspectives as part of the drug development process;
- Creation of a neurological disease surveillance program at the Centers for Disease Control (CDC); and
- Establishment of new incentives for studying and repurposing drugs for off-label for rare disease uses.
On September 18th, DM community leaders and their families from across the country visited with their Senators and Representatives urging action on the initiative. MDF is following up on this successful advocacy campaign and has announced the first ever DM Congressional Call-In on October 13th. All DM community leaders, their families, health care providers, and researchers – whether they participated in the MDF annual meeting in Washington, D.C. or not – are strongly urged to rally on behalf of Senate action on the bill.
To get involved, click here.