Building on Our Rare Disease Day Success - Advocating for Increased Myotonic Dystrophy Research

Published on Fri, 05/14/2021

Myotonic Dystrophy Foundation advocates from across the country did their part as part of another highly successful annual Rare Disease Day celebration and call to action on Sunday, February 28th. Together, we raised awareness of the challenges and opportunities facing individuals with rare genetic conditions like myotonic dystrophy, and urged our local, state, and federal elected representatives to take action to help us realize our goals for cures, treatments, and improved medical care.

There has been a lot of activity in Washington, D.C. to report on since Rare Disease Day. There are also opportunities to continue our research advocacy.

In early March, President Biden signed into law the American Rescue Plan Act which includes $1.9 trillion in COVID-19 economic and health care relief and related program funding. For Americans who receive their health insurance coverage through marketplaces established under the Affordable Care Act “Obamacare”, it expands income eligibility and increases the amount of financial support available to individuals and families to purchase health insurance. 

For individuals and families who lost their employer-based health insurance coverage and are relying on COBRA, it provides for temporary COBRA premium subsidies for up to 6 months during 2021. Subsidies will cover 100% of the monthly cost of COBRA while people are eligible. Finally, it includes new federal subsidies to encourage states to expand Medicaid health insurance eligibility.

In April, President Biden released his fiscal year 2022 federal budget. The president has proposed increasing NIH funding by $9 billion, to a total of $51 billion. His budget also seeks to launch the Advanced Research Projects Agency-Health (ARPA-H) at the NIH which would be modeled after the Department of Defense’s (DoD) Defense Advanced Research Projects Agency (DARPA). ARPA-H proponents cite DARPA’s success pioneering defense and civilian advancements like stealth technology, the Internet, automated voice recognition, and GPS technology as a model for groundbreaking health research. ARPA-H would be charged with accelerating the translation of basic scientific discoveries into new treatment and cures. 

Congress has begun the budget process by holding hearings on the President’s budget request and drafting legislation to fund the federal government for the next fiscal year. MDF and our advocates are working with Congress, Republicans and Democrats, to urge support for our federal research priorities. 

We are asking Congress to increase NIH and myotonic dystrophy research funding and to include myotonic dystrophy as an eligible research category in the Department of Defense (DoD) Peer Review Medical Research Program (PRMRP) for the 5th year in a row. 

Since our founding over 14 years ago, a growing and vocal group of advocates have made a difference in helping to increase NIH supported myotonic dystrophy research by $150 million and adding another $5.5 million in myotonic dystrophy funding as part of the PRMRP. As Congress begins to advance their budget bills, which will set the country’s funding priorities, we ask all MDF advocates to contact their Senators and Representatives to urge them to fully fund the NIH and continue to include myotonic dystrophy in the fiscal year 2022 PRMRP. Stay tuned for further updates and information that will help you reach out to Congress so we can fund even more research to better understand what causes myotonic dystrophy, discover improved treatments, and a cure. 

Click here to read more about MDF's advocacy.