News

Terapia Génica ELEVIDYS: Actualización Comunitaria Más Reciente con Sarepta Therapeutics Watch HERE The post Terapia Génica ELEVIDYS appeared first on CureDuchenne.

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 The post FDA fast tracks Dyscorban for treating heart problems in Duchenne appeared first on Muscular Dystrophy News.

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… The post Our 2026 Advocacy Agenda appeared first on Quest | Muscular Dystrophy Association.

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 The post My emotions blurred as I watched my play from ‘The Other Side’ appeared first on Muscular Dystrophy News.

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… The post MDA Ambassador Guest Blog: Pursuing My Dreams While Living with a Rare Disease appeared first on Quest | Muscular Dystrophy Association.

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, The post MDA-led project to map how human muscles repair themselves appeared first on Muscular Dystrophy News.

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 The post Conoce a Rosalia Sandoval Garcia appeared first on CureDuchenne.

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 The post My willingness to educate others about MD benefits all of us appeared first on Muscular Dystrophy News.

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… The post Simply Stated: Understanding Myotonia Congenita appeared first on Quest | Muscular Dystrophy Association.

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 The post The unbearable weight of grief that smoothes the jagged edges appeared first on Muscular Dystrophy News.

Three years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in a clinical trial, boys with Duchenne muscular dystrophy (DMD) are showing sustained improvements in physical function compared with what would be expected without treatment, according to new data announced by Sarepta Therapeutics, Elevidys’ developer. “ELEVIDYS is the first gene therapy for Duchenne to show The post New analysis tracks Elevidys outcomes 3 years after treatment appeared first on Muscular Dystrophy News.

In my previous column, I shared my concern about losing more strength due to the progression of my facioscapulohumeral muscular dystrophy (FSHD). This loss was making it increasingly difficult for me to get out of my friend Richard’s Subaru. Richard, who is also a musician, not only drives me to many of my musical performances The post Still playing my tune with a little help from my friends appeared first on Muscular Dystrophy News.

I’ve often written about learning to live alongside uncertainty. Duchenne muscular dystrophy has a way of quietly shaping plans, then loudly interrupting them. Last July, that disruption arrived as burnout during rehearsals for my graduating showcase at BEYOND DIS:PLAY, a performing arts training program in Singapore for disabled artists at ART:DIS, the country’s leading arts The post I define my story, even when Duchenne rewrites the script appeared first on Muscular Dystrophy News.

For people with Duchenne muscular dystrophy (DMD), 2026 promises to be a year of tremendous excitement, with several new therapies poised for likely approval in the U.S., according to DMD advocates. “It’s an exciting time,” Debra Miller, founder CEO of CureDuchenne, said in an interview with Muscular Dystrophy News Today. “A lot of hard work The post Advocates say 2026 will be ‘an exciting time’ for DMD community appeared first on Muscular Dystrophy News.

Every year, hundreds of thousands of people embark on a challenging journey to traverse the Camino de Santiago de Compostela, traveling days and weeks on foot to arrive in Santiago, Spain. The pilgrimage, which consists of multiple trails leading to the burial site of Jesus Christ’s disciple James, serves as a spiritual journey for many.… The post Making the Impossible Possible: One Man’s Journey on the Camino de Santiago de Compostela appeared first on Quest | Muscular Dystrophy Association.

PPMD is proud to partner with the Muscular Dystrophy Association (MDA), the FSHD Society, and the LGMD2L Foundation on a new, collaborative research grant focused on one of the most fundamental — and still unanswered… The post PPMD Joins Collaborative Research Effort to Decode How Human Muscle Regenerates appeared first on Parent Project Muscular Dystrophy.

Sarepta Therapeutics, Inc. has announced positive topline three-year functional results from Part 1-treated patients in EMBARK, the company’s global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals living with Duchenne. According… The post Sarepta Shares Topline Three-Year EMBARK Data for ELEVIDYS in Ambulatory Duchenne Patients appeared first on Parent Project Muscular Dystrophy.

Sarepta Therapeutics announced positive topline data from the EMBARK trial, showing that Elevidys, 3 years post treatment, significantly slows disease progression on functional measures  in ambulatory individuals who were 4-7 The post Sarepta reports positive 3-year data from the EMBARK trial:  4-7 year olds treated with their microdystrophin gene therapy, Elevidys, have significantly slower disease progression appeared first on CureDuchenne.

Solid Biosciences, Inc., has shared updates on the company’s gene therapy program for the treatment of Duchenne. According to Solid, 33 participants have now been dosed in the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial… The post Solid Biosciences Shares Update on INSPIRE DUCHENNE Trial Evaluating SGT-003 appeared first on Parent Project Muscular Dystrophy.

January doesn’t just bring a new calendar year. For four of us in this family, it’s when we celebrate our birthdays. A new year of life! I live in Nebraska with my husband and six of the seven children we share. My daughter, Lexi, 24, lives in another town with her husband, but Max, 20, The post A caregiver’s birthday offers an opportunity to practice self-care appeared first on Muscular Dystrophy News.

For over 70 years, MDA’s National Ambassadors have been an intrinsic part of championing for our mission to empower people living with neuromuscular disease and increase care, research, and advocacy efforts. Each year, these Ambassadors share their lived experiences, perspectives, and passion to raise awareness and create positive change for our community. Lily Sander, an… The post What’s Ahead for Your 2026 National Ambassador appeared first on Quest | Muscular Dystrophy Association.

Avidity Biosciences has released answers to frequently asked questions about their Managed Access Program (MAP) for individuals with Duchenne who are amenable to exon 44 skipping and meet clinical criteria. The post Avidity Biosciences Managed Access Program (MAP) – Frequently Asked Questions appeared first on CureDuchenne.

Solid Biosciences reports that 33 children have now been dosed in their SGT-003 gene therapy trial for Duchenne. So far, the treatment has been generally well tolerated with signs of improved muscle The post Solid Biosciences Reports Progress on Next-Generation Gene Therapy  appeared first on CureDuchenne.

In this Quest Podcast episode, we chat with public speaker, consultant, and disability advocate, Jax Cowles. Jax shares an honest, thoughtful, and deeply creative conversation about daily life, independence, and problem-solving.  She opens up about how creativity and “life hacking” became essential tools rather than optional skills, and how small, low-cost adaptations can completely transform… The post Quest Podcast: Redesigning the Day: Accessibility and Mindset Life Hacks with Jax Cowles appeared first on Quest | Muscular Dystrophy Association.

Capricor Therapeutics, which received early funding from CureDuchenne, said the FDA has requested the full clinical study report from their latest clinical trial, the HOPE-3 trial.  Positive topline results from The post Capricor provides regulatory update on Deramiocel for Duchenne appeared first on CureDuchenne.