Our family has encountered many challenges in our journey with Duchenne muscular dystrophy, and there will be more to come. If I were to ask the question, “What is the hardest part of Duchenne?” in a room full of parents whose children have the condition, there likely would be as…
Photo courtesy of Matt DeEmilio Day 2 of 30 This is Matt DeEmilio’s story: My name is Matt DeEmilio. I’m 27 years old, and I am the coordinator for disability support services at Coastal Carolina Community College in North Carolina. My job entails granting reasonable accommodations for equal access,…
A myriad of activities are afoot to mark National Muscular Dystrophy Awareness Month, observed each September. The events aim to raise funds for, and heighten awareness of, muscular dystrophy (MD) and related neuromuscular disorders thought to affect more than 300,000 families across the U.S. Awareness days are set for…
Photo courtesy of Ross Hovey Day 1 of 30 This is Ross Hovey’s (@thesmartchap_rosshovey) story: Would I have preferred a life without disability? The simple answer to this question is “Yes.” But do I grudge having spinal muscular atrophy? Not really, as I don’t know anything different. I think…
I started writing for my high school newspaper, The Bolt, when I was 17. Now 24, I’m telling the stories of people who make an impact in their rare disease community for Bionews, the publisher of this site. Over the last seven years, I’ve had the privilege of conducting…
The European Commission (EC) has granted orphan designation to AOC 1001, Avidity Biosciences’ investigational therapy for myotonic dystrophy type 1 (DM1). This designation is given to investigational therapies that have the potential to treat rare, life-threatening, or very serious diseases that affect less than five in 10,000 patients…
It’s tempting to label the siblings of those who have Duchenne muscular dystrophy as the “unaffected siblings.” But I can never seem to get those words out of my mouth. Siblings are indeed affected by Duchenne. In my family, the siblings, Lexi, Chance, and Mary, experience Duchenne daily, even though…
Cumberland Pharmaceuticals is recruiting boys and men for a Phase 2 study evaluating the safety, efficacy, and duration of effect of its oral ifetroban therapy for cardiomyopathy — a disease of the heart muscle — linked to Duchenne muscular dystrophy (DMD). The study, called FIGHT DMD (NCT03340675),…
MRI scans of muscle can be used as a biomarker to assess disease progression in Duchenne muscular dystrophy (DMD) and to support the evaluation of different therapies in clinical trials, an analysis showed. “The results of this study provide further support for the use of imaging as an objective,…
When I was a toddler, my parents would sit me up in my highchair in front of a big bowl of broccoli. It was around dinner time, while they were still preparing the food, so I was a very hungry baby. I could only eat the rest of the meal…
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