Tell Congress to Protect and Increase NIH Funding for Myotonic Dystrophy Research
Congress has not yet passed a full-year Fiscal Year 2026 budget for the U.S. Department of Health and Human Services, which includes critical funding for myotonic dystrophy (DM) research supported by the National Institutes of Health. Without a full-year budget, Congress risks harmful cuts that would slow scientific progress and delay discoveries that could improve the lives of individuals and families living with myotonic dystrophy. There is a real possibility that Congress may approve a budget with reduced federal biomedical research funding or fail to pass a full-year budget at all. Click here to urge Congress to support myotonic dystrophy research and care!
This year, NIH funding has already been reduced by 2.5 billion dollars through program reductions or suspensions, with nearly 2,500 scientific grants frozen or terminated. (Source.) Myotonic dystrophy researchers report significant delays and setbacks as a result of these disruptions.
Myotonic dystrophy research is at a pivotal stage. NIH investments power the discoveries and clinical trials that thousands of individuals and families living with this complex, multi-systemic disease depend on. Any further cuts will slow scientific progress and delay future treatment options. Send Your Message to Congress Now – Every Email Makes a Difference!
Learn How You Can Be an MDF Advocate
Do you believe the US Congress should do more to support DM research? You can make a difference!
Watch MDF’s 2025 State Advocacy Training webinar to learn about MDF’s advocacy priorities, the role of U.S. State Advocacy Captains, and simple ways to advocate for DM research funding at the federal level.
Watch our US State Advocacy Captain Training Webinar!
Raise Your Voice
The myotonic dystrophy community must raise our voices to create change! When we speak up together, we influence federal research funding, the development of new therapies, clinical trial efforts, and initiatives that improve the quality and affordability of care.
Despite the urgent needs of people living with DM, federal funding for myotonic dystrophy research still ranks near the bottom of federally funded disease research. Today, the National Institutes of Health (NIH) invests approximately $11 million annually in DM research, out of a total budget of $43 billion.
How MDF Advocates Are Expanding Federal Research Funding for DM
$26.5 Million Secured for DM Research Through PRMRP
For nine years in a row, MDF has successfully advocated for DM’s eligibility for federal research funding through the Peer Reviewed Medical Research Program (PRMRP). This advocacy has resulted in DM researchers securing $26.5 million in competitive research grants, significantly improving our understanding of the causes of DM and identifying new ways to improve treatment and care.
Congress established the PRMRP program in 1992 to advance novel and groundbreaking approaches to biomedical research.
Read more about PRMRP-funded DM research.
New Federal Support for DM Through the Repeat Expansion Disease Initiative (REDI)
MDF Advocates secured congressional support for a provision in the Fiscal Year 2023 budget directing the NIH to establish the Repeat Expansion Disease Initiative (REDI). This initiative increases scientific focus on repeat expansion diseases like DM and encourages the NIH to consider new funding mechanisms across multiple institutes to support discoveries that lead to treatments and cures.
This marked the first time Congress has recognized the rapidly emerging science on DNA repeat expansions, which causes more than 50 distinct diseases, including myotonic dystrophy, Huntington’s disease, and other neurological conditions. Congress identified DM as a model for this class of diseases caused by repeat instability and toxic RNA and directed the NIH to advance REDI to support cutting-edge research.
MDF continues to engage with Congress and NIH leadership to leverage the scientific opportunities created by REDI and accelerate new treatments for DM and related genetic conditions.
Learn more about the impact of the Repeat Expansion Disease Initiative (REDI).
Progress Toward the First FDA-Approved Treatment for DM
In 2016, MDF hosted the first-ever Patient Focused Drug Development meeting with the FDA to share patient insights and data used to guide the review and approval of future DM therapies. Prior to this groundbreaking meeting, very few companies were investing in DM drug development.
Today, over 20 companies are pursuing drug candidates for myotonic dystrophy, with several therapies expected to move toward FDA review in the coming years.
MDF works closely with biopharmaceutical companies and regulatory agencies, including the Food and Drug Administration (FDA) and the European Medicines Agency (EMA), to bring the voices of people living with DM into the drug development and approval process. This helps ensure that new treatments address real needs and offer meaningful benefits, while also supporting future advocacy for insurance coverage once these therapies become available.
At the 2025 MDF Conference, many companies shared updates on clinical trials and their progress toward these promising DM treatments.
Watch Industry Updates from the 2025 MDF Conference.
Get Started!
Join the Myotonic Dystrophy Foundation's advocacy program and add your voice to efforts that improve care, advance research, and raise DM awareness in Congress, federal and state agencies, the medical community, and the media.
Advocacy 101
What is Grassroots Advocacy?
Grassroots advocacy is when individuals come together to educate elected officials about the needs of their communities and ask for support. When people who share common goals speak up collectively, they can influence funding decisions and public policies that affect the lives of people across the country, including those living with DM.
Why Should I Become an Advocate?
You have a voice and a vote, and they matter! When members of Congress hear directly from their constituents, they better understand what issues are important to the people they represent. If they never hear from the DM community, they may not realize how urgently research funding and supportive policies are needed.
Your outreach can influence how a Senator or Representative approaches a vote or chooses to support DM-related priorities. Advocacy is one way you can help shape decisions that improve the lives of people living with DM.
Can One Person Really Influence Congress?
Yes. Members of Congress pay close attention to the views of their constituents. While they may have established positions on major national issues, many are open to learning more about conditions like myotonic dystrophy and the needs of the communities affected.
Your voice could help educate an elected official, build a new relationship, or even inspire a long-term champion for DM research and related policy goals.
Your Voice Makes a Difference
We need your help to show Congress why these issues matter to the DM community and to build relationships with your elected officials and their staff. You don't need to be in Washington to make an impact. You can take action from home by sending an email, making a phone call, or requesting a meeting.
If you're ready to get started or need support along the way, please contact MDF at 415-800-7777 or info@myotonic.org.
Make a Phone Call
Taking a minute to call your representatives can make a difference for people living with DM. Tell your elected officials about your connection to DM and ask for their support for one of MDF's congressional priorities, such as increased federal funding for DM research.
When you write or speak to them or their staff, be sure to identify yourself as a constituent by sharing your name and address. Contact information, including addresses, phone, numbers can be found through www.congress.gov or by calling the US Capitol Switchboard at 202-234-3121.
Visit a District Office
Another effective way to advocate is by requesting a meeting with your elected official and their staff at a local district office (refer to the House of Representatives or Senate directories). These meetings help build awareness and strengthen relationships over time.
Before your meeting, we recommend contacting MDF to help you prepare. We can update you on our current advocacy priorities, help you feel confident telling your story, and support follow-up efforts to reinforce the importance of DM research funding.
Advocacy Resources
Below are resources to help you feel prepared and confident as an advocate. If you have questions or need help with any advocacy related activities, contact MDF at 415-800-7777 or info@myotonic.org.
Rare Disease Day Resources
- Watch the 2024 Rare Disease Day Webinar for guidance on building relationships with elected officials and advancing research funding goals.
- View MDF's 2023 Rare Disease Month Advocacy Workshop to learn more about MDF's advocacy efforts during this important month of awareness and action.
- Review MDF Congressional Advocacy Talking Points for tips on contacting Congress and what to say during meetings.
- Explore MDF Advocacy Priorities, a background document outlining MDF's congressional requests to share with congressional staff.
- Use the Sample Letter to Congress if you prefer to email or send a written message.
- Become a State Advocacy Captain by watching MDF's webinar with step-by-step guidance on helping increase DM research funding.
Advocacy Glossary
Government and legislative language can feel overwhelming at first. Our advocacy glossary explains commonly used terms in the legislative process to help you feel more comfortable and informed.
MDF Advocacy Webinars & Videos
MDF offers advocacy webinars throughout the year that explain current issues and campaigns. Advocacy training is also provided at our annual conference, including sessions such as FDA 101.
Find past webinar recordings on the MDF Digital Academy.
Other Resources:
- Register to vote to make your voice heard on issues that matter to you and the DM community.
- Learn about bills and other legislation via Congress.gov, the Library of Congress's official web portal.
- Visit Project Vote Smart to see how your Senators and Representatives have voted on specific issues.