-
Wildland Fire Fighter Refuses to Allow Diagnosis to Define His Life
When Tyler Long was diagnosed with facioscapulohumeral muscular dystrophy (FSHD) during his senior year of high school, doctors advised him that this diagnosis meant that he should plan his future around a “non-strenuous” lifestyle. For the active and athletic outdoorsman, this suggestion wasn’t in alignment with how he saw himself and how he wanted to…
-
Sarepta plans to seek full FDA approval of 2 Duchenne treatments
Sarepta Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to grant full approval for Amondys 45 (casimersen) and Vyondys 53 (golodirsen), two exon-skipping therapies designed to treat certain people with Duchenne muscular dystrophy (DMD). The company said it met with the FDA to discuss plans for the two therapies, and the FDA…
-
PPMD Provides $250,000 in Funding to Secretome Therapeutics Through PPMD Venture Pathways Program to Support Development of Cardiac Cell Therapy
PPMD and Secretome Therapeutics (Secretome) are excited to announce that PPMD has provided $250,000 in funding to Secretome through PPMD Venture Pathways, our venture-philanthropy initiative that provides funding to industry to accelerate therapeutic development for Duchenne and Becker. This funding commitment aims to advance the development of STM-01, Secretome’s neonatal cardiac progenitor cell (nCPC) product…
-
When it comes to rare disease advocacy, just keep showing up
According to the EveryLife Foundation for Rare Diseases, more than 30 million Americans live with at least one rare disease. Considering that’s nearly 10% of the U.S. population, these diseases don’t seem so rare after all. For decades, the disability rights movement has fought for the rights of this population, advocating for greater accessibility and…
-
MDA 2026: Dyne advances DMD therapy z-rostudirsen toward approval
Dyne Therapeutics is advancing its investigational exon-skipping therapy zeleciment rostudirsen (z-rostudirsen), formerly known as DYNE-251, toward regulatory approval after trial data showed early signs of benefit across multiple disease measures in boys with Duchenne muscular dystrophy (DMD). The most recent data from the Phase 1/2 DELIVER clinical trial (NCT05524883) showed that the therapy increased levels of…
-
MDA 2026: This year’s MDA meeting provided a glimpse into the future
Muscular Dystrophy News Today is wrapping up coverage of the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, which ran from March 8-11 in Orlando, Florida, and online. This year, more than 2,400 attendees from 40 countries gathered in person and virtually to discuss recent advances in neuromuscular disease research and care — and…
-
How to Make Vocational Training Affordable
There are many reasons a person with a disability might choose vocational training. One prominent reason: It can provide an affordable, direct pathway to employment, making it an attractive option for anyone interested in entering the workforce. Vocational training, also called trade school or technical education, provides training targeted to a specific career. Unlike traditional…
-
I’m thankful for a big family that gives my DMD sons a full social life
The comment comes in many forms: “I don’t know how you do it.” “You have your hands full!” “I can’t imagine what it would be like.” It’s always in response to seeing my large family. I share seven children with my husband, Jason: Lexi, 24; Max, 20; Chance, 18; Rowen, 17; Charlie, 15; Mary, 11;…
-
17-year-old advocate shares her NMOSD story to raise awareness
In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To cope with all the changes in her life,…
-
MDA 2026: ‘Body of evidence for Elevidys continues to grow’
Boys with Duchenne muscular dystrophy (DMD) who received Sarepta Therapeutics’ gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in a clinical trial continue to show signs of slowed disease progression relative to DMD’s natural course, along with caregiver-reported improvements in everyday living. That’s according to data from the Phase 3 EMBARK clinical trial (NCT05096221) presented at the 2026…
