The Muscular Dystrophy Association (MDA) has awarded more than $17 million to projects aimed at advancing research and developing new treatments in muscular dystrophy (MD) and other neuromuscular diseases. The funding adds to the more than $1 billion the MDA has already invested in research. “The Muscular Dystrophy…
There is power in community! I’m thankful that my family is part of a big, beautiful community: the parents, professionals, and, of course, those living with Duchenne muscular dystrophy (DMD). They are, in my opinion, the best! When my three sons — Max, Rowen, and Charlie, ages 16, 13,…
Four months of treatment with the experimental oral therapy EDG-5506 reduced markers of muscle damage and improved measures of physical function among men with Becker muscular dystrophy (BMD) in the Phase 1 ARCH clinical trial, new data show. Edgewise Therapeutics, the company developing EDG-5506, recently launched a Phase…
The first patient has received Dyne Therapeutics‘ investigational therapy DYNE-251 for treating Duchenne muscular dystrophy (DMD), the company has announced. The therapy is being tested in the DELIVER Phase 1/2 clinical trial and is indicated for patients with mutations amenable to exon 51 skipping. “We are excited to…
A neuropsychological evaluation greatly affected how we plan to educate our 13-year-old son this school year. As Rowen begins the eighth grade, I am proud of the services he will receive. A neuropsychologist hasn’t always been a part of the care team for our three sons with Duchenne…
Vamorolone, an investigational therapy for Duchenne muscular dystrophy (DMD), maintained biomarkers of bone formation and turnover compared to prednisone, according to the now published details of the VISION-DMD study. Standard anti-inflammatory corticosteroids, such as prednisone, have been shown to stunt growth with long-term use. Vamorolone is a…
Treatment with Exondys 51 (eteplirsen) outperformed standard of care therapy at delaying respiratory decline in boys with Duchenne muscular dystrophy (DMD), an analysis of data from several clinical trials found. Compared with boys on other therapies, those treated with Exondys 51 in trials had “a delay of…
If you thought you missed my column last week, it wasn’t you — I didn’t write one. My kids were sick with cold symptoms. One thing you may not know about large families is that germs spread like wildfire, and the entire family gets whatever is going around. There are…
For the fourth year, a family in Montana is hosting “Calves to Cure,” a cattle auction that aims to raise funds to support developing new treatments, or even a cure, for Duchenne muscular dystrophy. The event will take place Sept. 15 in Billings, Montana. All proceeds from the…
As a person with Duchenne muscular dystrophy, I’ve always felt socially deprived and isolated. It’s been an extremely painful and lonely journey for me. Many of us with Duchenne also have undiagnosed anxiety and depressive disorders, adding layers of complexity to our already arduous paths. Most…
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