The seventh annual Napa in Newport wine auction, an event that brings together acclaimed Napa Valley vintners, raised more than $1.5 million for CureDuchenne. The nonprofit will use those proceeds to support people with Duchenne muscular dystrophy (DMD), the most common form of MD, and to find…
The title of this column comes from a meme I saw this week. Its message found me at a time when I needed to hear it. My three sons with Duchenne muscular dystrophy have appointments at Children’s Hospital Colorado the first week of December. (Yes, we must take three…
A Phase 1 trial of SRP-9001, Sarepta Therapeutics‘ investigational gene therapy, is recruiting 3-year-old boys with Duchenne muscular dystrophy (DMD) who are able to walk for a new patient group in California, according to a press release from the Muscular Dystrophy Association. This group, being enrolled at the…
Upper limb strength, function, and muscle fat fraction continuously worsen over time in Duchenne muscular dystrophy (DMD) patients with mutations amenable to exon 53 skipping, according to three-year data from a natural history study. Notably, this progression of upper limb involvement was assessed reliably with strength, function, and muscle…
We form our identity from the world around us. That includes factors like our relationships with parents, siblings, and friends, what we look like, our sexual orientation, and our religious beliefs, among others. For me, one unspoken taboo is my disability. I use a wheelchair because I can barely walk,…
Halloween is over, and I’m happy to say we all got through it. While it was one of my favorite holidays when I was a kid, celebrating it with children with Duchenne muscular dystrophy has made trick-or-treating challenging in ways I never dreamed of. This week, I’ll be writing…
The 2021 holiday retail fundraising program to support the Muscular Dystrophy Association (MDA)Â recently opened. The multiple retailers involved in this year’s campaign hope to raise at least $500,000 to support research and care for people with muscular dystrophy, amyotrophic lateral sclerosis (ALS), and related neuromuscular diseases.
Welcome back to “Hidden Truths,” a column that seeks to offer an honest look at living with a neuromuscular disease. I not only like to present my own experiences, but also to challenge others to examine themselves and think outside the box. I believe that by questioning and searching for…
A Phase 1 clinical trial evaluating Edgewise Therapeutics’ experimental oral therapy EDG-5506 in healthy volunteers and men with Becker muscular dystrophy (BMD) has dosed the first BMD patients. The study (NCT04585464) is currently recruiting up to eight men, ages 18 to 55, with BMD at its…
I have shared many times in this column that I have three sons with Duchenne muscular dystrophy. People sometimes think I’m an expert on the subject. But the truth is that my boys are unique, and Duchenne has affected each of them differently. Their disease progression and weakening muscles…
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