On April 25, the Muscular Dystrophy Association (Singapore), or MDAS, celebrated its 25th anniversary with a spectacular gala at Marina Bay Sands, an iconic hotel and convention center here in Singapore. The evening was filled with music, memories, and meaningful reunions. The staff had adorned the room, which quickly filled…
The U.S. Food and Drug Administration (FDA) is on track to make a decision about deramiocel, an experimental cell therapy designed to treat heart disease in Duchenne muscular dystrophy (DMD), by the end of August. The FDA agreed this year to review an application to approve deramiocel for…
Living happily with limb-girdle muscular dystrophy, as I do, requires patience, a positive attitude, and a good support system of family, friends, and a professional care team. But I’ve learned there’s another huge component for me to succeed: planning. Abilities that able-bodied people take for granted often require well-thought-out…
I am going at a pace I can’t maintain. As a former half-marathon runner, I can feel the burn in the pit of my stomach as I write this. I can feel the heat in my legs and cheeks as I push myself to the finish line. Except this time,…
People with myotonic dystrophy (DM) use more healthcare resources and face higher associated costs than patients without the disease, according to a U.S. study that analyzed a large database of insurance claims. After diagnosis, DM patients are more likely to be hospitalized and visit an emergency department, researchers found.
In a recent column, I talked about paring down my music rig and retiring my accordion. Over the years, I’ve transitioned from a 120-bass professional accordion to a 32-bass student model that, while less than 15 pounds, is now too heavy for me to handle. Because of my facioscapulohumeral…
The Committee for Medicinal Products for Human Use, an arm of the European Medicines Agency known as CHMP for short, has recommended the approval of Duvyzat (givinostat) as a treatment for people with Duchenne muscular dystrophy (DMD), ages 6 and older, who are able to walk. The European…
I’m writing this column as my husband, Jason, drives our family through the last rolling hills on the eastern side of the Rocky Mountains. Last week, we took an epic road trip from our quiet hometown in Nebraska to the eclectic sounds and neon lights of the Las Vegas Strip.
CureDuchenne will host its annual Futures National Conference May 22-25 in San Antonio, as part of an effort to provide education and offer connections to members of the Duchenne and Becker muscular dystrophy communities. Under the theme “Together We Thrive,” the event will share information on research and…
Two days ago I marked the second anniversary of my relationship with my girlfriend, Amanda. As I celebrated this milestone, I found myself reflecting deeply on how much our lives have changed since we became a couple. Duchenne muscular dystrophy (DMD), my lifelong companion, has undoubtedly influenced…
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