Presented on September 8th, 2023.

Diana Varela, Public Affairs Specialist
Social Security Administration

Joyce Lee, Medical Relations Officer
Department on Disability Services

Gain a better understanding of the role of the Social Security Administration (SSA) programs, benefits, and services available to the DM community in the US. Leaders from the SSA will join this panel to help demystify the application, denial, and appeal process.

Click here to learn more about the 2023 MDF Annual Conference.

Presented on September 15, 2022.

Join MDF on International Myotonic Dystrophy Awareness Day on September 15th to learn how advocates can increase #myotonicDystrophy (DM) awareness, grow DM research funding, and advance policies to accelerate the approval of drugs to treat and eventually cure DM.

As Congress advances spending legislation that includes biomedical research funding, we will be closely following these proceedings, and as we identify opportunities for MDF advocates to influence this legislation, we will be urging advocates to follow-up with their US Senators and Representatives.

Click here to learn more about the success of MDF's first Advocacy Week and the critical need to continue federal funding of DM research.

Click here to find more myotonic dystrophy advocacy resources.

Presented on September 10, 2022.

Representatives from biotech and pharma companies will provide updates on their drug development efforts in the DM field. Presenters include:

• PepGen
• Juvena Therapeutics
• Entrada Therapeutics
• NeuBase Therapeutics
• ARTHEx Biotech

Click here to learn more about the 2022 MDF Annual Conference.

Presented on September 9, 2022.

Representatives from biotech and pharma companies will provide updates on their drug development efforts in the DM field. Presenters include:

• Harmony Biosciences
• Avidity Biosciences
• Dyne Therapeutics
• GrittGene Therapeutics
• AMO Pharma Ltd.

Click here to learn more about the 2022 MDF Annual Conference.

Learn about the different resources that have been created for the Myotonic Dystrophy community! This presentation provides an introduction to help familiarize you with the resources and support options available for families affected by myotonic dystrophy.

The resources discussed during this video can be found at:

• Clinical Care Documents, Guidelines, and Toolkits
• Study and Trial Resource Center
• Request Warmline Phone Support
• Find a Myotonic Dystrophy doctor near you
• US Financial Resources
• Juvenile-onset Adult (JOA) Programs
• Digital Academy with recordings of previous conference sessions and webinars
• Myotonic Dystrophy Support Groups

For newly diagnosed families, we recommend the following resources:

• Start your myotonic dystrophy journey here
• Sign up for the Myotonic email list
• Learn about the impacts of myotonic dystrophy with the Body Systems Tool
• Confirm you diagnosis with a Genetic Test
• Get answers to Frequently Asked Questions (FAQs)

Have questions? Need more help? Contact us!

Representatives from biotech and pharma companies will provide updates on their drug development efforts in the DM field, including companies new to the Myotonic Annual Conference. The industry updates are followed by the Q&A session where questions submitted throughout the conference are answered by a panel of DM Professionals.

Matt Disney, PhD., Expansion Therapeutics - Slides
Art Levin, PhD, Avidity Biosciences - Slides coming soon!
Laury Mignon, PhD, Ionis Therapeutics - Slides coming soon!
Mo Qatanani, PhD, Dyne Therapeutics - Slides

Heart Health: Understanding DM Cardiac Symptoms with Saman Nazarian, MD, PhD

This session explains how myotonic dystrophy may impact your heart and provides an overview of the cardiac electrical system, common symptoms associated with conduction problems, and preventative measures.

Download the Heart Health Slide Deck.

Join Certified Genetic Counselor Tiffany Grider from the University of Iowa for an recorded webinar on genetic testing. Topics include:

+ The underlying genetic cause of myotonic dystrophy type 1.
+ How this genetic cause leads to so many different medical problems including muscle weakness.
+ Inheritance, the risks for congenital myotonic dystrophy and the genetic basis for the more severe symptoms.
+ Different types of genetic testing including diagnostic, presymptomatic, and prenatal.
+ Research being done for gene therapies.

View Tiffany Grider's clinical profile.

Download the slides (.pptx).

MDF created a short video to recognize and celebrate Moxley’s incredible commitment and impact on the treatment of myotonic dystrophy and scientific discovery to drive disease understanding and therapy development. “Mox” is in many ways irreplaceable, functioning as a singular force in the myotonic dystrophy field for over four decades.

Read more about the tribute to Dr. Moxley.

There are many heroes in the myotonic dystrophy community, from the people living with this disease to their caregivers, physicians and the researchers who focus on developing therapies and better disease understanding every day. Watch this moving short film to hear a few of these heroes described and celebrated.