On February 14, 2025, the RNA Institute and the Myotonic Dystrophy Foundation (MDF) organized and co-hosted the first ever symposium exploring how to include myotonic dystrophy (DM) in newborn screening. Conditions included in newborn screening are also diseases that do not have treatments but benefit from early disease management thereby significantly improving clinical outcomes.
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The Latest
A recent publication in Nature Genetics explores base editing as a solution to repeat expansion diseases similar to myotonic dystrophy (DM). This summary was created by Asmer Aliyeva, PhD Candidate in Dr. Andy Berglund’s lab at the RNA Institute, in collaboration with MDF's Chief Scientific Officer, Dr. Andy Rohrwasser.
This summer, a cross-country journey is capturing hearts and uniting motorcycle enthusiasts, rare disease advocates, and families impacted by DM. Patrick "Pat" Cornell—a 60-year-old Navy veteran, 9/11 first responder, and retired firefighter—is riding 125,000+ miles in 125+ days to raise awareness and funds for MDF!
For the second year in a row, thanks to the work of MDF advocates across the country, 6 U.S. Senators signed Senator Amy Klobuchar’s (D-MN) letter to the Senate Appropriations committee in support of our request for $10 million in new DM research funding as part of the Congressionally Directed Medical Research Program.