PGN-EDODM1, Pepgen’s experimental therapy for myotonic dystrophy type 1 (DM1), was generally well tolerated in an early clinical trial, with biomarker data suggesting that the treatment is affecting its intended molecular target. Based on these early…
Discussion
My facioscapulohumeral muscular dystrophy (FSHD) has progressed to the point where daily activities are increasingly challenging, and often impossible, to manage on my own. As a result, I now need more help than ever before. I know I’m doing the best I can. I exercise daily, hoping to keep…
Becker muscular dystrophy (BMD) patients who received the experimental treatment sevasemten in clinical trials had stable motor function over several years of follow-up, new data showed. That stands in contrast to the typical progression of BMD, in which motor function steadily declines as the disease progresses. The data…
At this year’s Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, researchers and clinicians are talking about the central role of patients, families, and caregivers in driving change for people living with neuromuscular diseases. Muscular Dystrophy News Today sat down with John F. Crowley, president and CEO of the…
Discussion
Living with a rare disease is incredibly complex, with countless layers to navigate. Managing daily life amid so many unknowns — and trying to find a regimen that truly works — can feel overwhelming. From the routine of medications and other relentless therapies to countless medical frustrations, we’re forced…
Becoming educated and informed about muscular dystrophy is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your muscular dystrophy journey.
There is no cure for muscular dystrophy yet, but here you can find more information about current therapeutic approaches, as well as experimental treatments that are in the pipeline.
Get regular updates to your inbox.
