Current Therapeutic Approaches | Myotonic Dystrophy Foundation

Agenda Category

Closed Professional Session

Agenda Date Time

Fri, 09/13/2019 - 10:00AM - Fri, 09/13/2019 - 12:00PM

Agenda Chairs

Moderator: Douglas Kerr, MD, PhD, MBA, Generation Bio

Agenda Location

Professional

Agenda Context

2019 MDF Annual Conference

Agenda Detail

Agenda Detail Item

Current Therapeutic Approaches moderated by Douglas Kerr, MD, PhD, MBA, Generation Bio.

Andrew Berglund, PhD, RNA Institute
Vincent Dion, PhD, Cardiff University School of Medicine
Matthew Disney, PhD, Scripps Research Institute
Steve Finkbeiner, MD, PhD, Gladstone Institutes
Darren Monckton, PhD, University of Glasgow
Christopher Pearson, PhD, Hospital for Sick Children
Romesh Subramanian, PhD, Dyne Therapeutics
Eugene Yeo, PhD, UCSD/Locana Bio

Agenda Detail Time

10:00am

To Time

12:00pm

Myotonic Dystrophy News

July 23,2025

Could Myotonic Dystrophy Be Included in Newborn Screening? Experts Say It’s Time to Explore

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.

July 23,2025

Fixing DNA Typos: New Hope for Myotonic Dystrophy

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.

June 27,2025

125,000+ Miles for Awareness: One Man’s Ride to Shine a Light on Myotonic Dystrophy

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!

June 27,2025

Thank You Advocates, 6 U.S. Senators Sign DM Research Letter!

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.

June 27,2025

Meet MDF's Summer 2025 Interns!

MDF is excited to highlight the work of two MDF interns, who are contributing to MDF's mission of Community, Care, and a Cure this summer! We appreciate all of their enthusiasm to improve our support of the DM community!

Questions?

Request Warmline Support! MDF offers a professionally staffed Warmline for people living with myotonic dystrophy, their caregivers, medical professionals and others to ask questions, find support and guidance and, when appropriate, be connected to additional resources and services. Click here to request Warmline Support.

Additional questions? Contact MDF by email at info@myotonic.org or give us a call at 415-800-7777.