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Our 2026 Advocacy Agenda
After a year of twists and turns in 2025, MDA and its advocates are even more motivated to raise their voices and create change for the neuromuscular community. Check out the roadmap for the next 12 months and learn how you can make an impact in 2026. Celebrating early victories on previous priorities 2026 got…
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FDA fast tracks Dyscorban for treating heart problems in Duchenne
The U.S. Food and Drug Administration (FDA) has granted fast track designation to Dyscorban (ifetroban), Cumberland Pharmaceuticals’ treatment candidate for heart problems in Duchenne muscular dystrophy (DMD). Dyscorban has been tested in the Phase 2 FIGHT DMD trial (NCT03340675), with data showing that it improved heart function and reduced markers of heart damage in individuals with…
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Terapia Génica ELEVIDYS
Terapia Génica ELEVIDYS: Actualización Comunitaria Más Reciente con Sarepta Therapeutics Watch HERE The post Terapia Génica ELEVIDYS appeared first on CureDuchenne.
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MDA Ambassador Guest Blog: Pursuing My Dreams While Living with a Rare Disease
Gabrielle Runyon is a graduate student in the Master’s Counseling Program at the illustrious Tennessee State University. She is from Louisville, Kentucky. Gabrielle was diagnosed with spinal muscular atrophy (SMA) when she was one year old. Her fun fact is that she can play three instruments. Living with a rare disease, I learned early on…
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My emotions blurred as I watched my play from ‘The Other Side’
In my previous column, I shared my experience of stepping away from performing in my own play, “The Other Side,” and trusting that the work could continue without me at its center. This column picks up where the last left off: the moment the play met its audience, and I found myself watching from an…
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MDA-led project to map how human muscles repair themselves
A research collaboration led by the Muscular Dystrophy Association (MDA) aims to create a molecular map of human muscle regeneration, a project that could accelerate the development of muscle repair-based therapies for people with muscular dystrophy (MD). Abigail Mackey, PhD, a professor of muscle physiology and regeneration at Copenhagen University Hospital, will lead the project,…
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Conoce a Rosalia Sandoval Garcia
Rosalia es una orgullosa mamá de Joe (8), quien vive con Duchenne, y de Sophia (6). Ella misma vive con distrofinopatía, lo que hace que esta misión sea especialmente cercana a su corazón. Con 13 años de experiencia como terapeuta del habla bilingüe en el área de Austin, Texas, Rosalia siente una gran pasión por…
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My willingness to educate others about MD benefits all of us
After my muscular dystrophy diagnosis in August 1985, I thought I could hide my symptoms from my friends at school. When I started seventh grade a few weeks later, I was in a new building with kids from all over town, not just my neighborhood. I was determined not to let any of them know…
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Simply Stated: Understanding Myotonia Congenita
Myotonia congenita (MC) is a rare, inherited neuromuscular condition characterized by muscle stiffness (myotonia) present during infancy or childhood. Unlike some other neuromuscular disorders, MC does not cause progressive muscle loss (atrophy). So, while people with MC often appear muscular, they may struggle with everyday movements. It has been estimated that MC affects approximately 1…
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The unbearable weight of grief that smoothes the jagged edges
I type my columns on Tuesdays, but I write them in my head in the days before. I consider what to write, how to begin, and the words to use to share my life experiences. My family is fun, and my columns often reflect that, to show the lessons I’ve learned and the positivity I…
