A new gene therapy approach safely restored the production of emerin, the protein missing in people with X-linked Emery-Dreifuss muscular dystrophy (EDMD), in both a mouse model and human muscle cells, according to a study. Although the preclinical study could not assess the gene therapy’s impact on muscle function,…
Discussion
We had a medical scare last week, and we handled it like professionals. I hate that about us. The day started like most days for our big family. I took our daughter Callie to preschool, and then, because it was fall break, our son Charlie had band practice at 9…
Discussion
Dyne Therapeutics‘ experimental therapy zeleciment basivarsen (z-basivarsen) may help improve multiple measures of physical function in people with myotonic dystrophy type 1 (DM1), new clinical trial data suggest. “What’s encouraging is that we observed improvements across multiple measures, including strength and functional assessments,” Doug Kerr, MD, PhD, chief…
Discussion
Last Friday, my girlfriend, Amanda Yip, and I celebrated the second year of Rebirth Ensemble, the disability-led creative business we founded in Singapore in October 2024. We began with an ambition: to make art more accessible. Now, we’re exploring creative storytelling, multisensory art, and accessibility consultancy. Yet the biggest change…
Discussion
Mental health problems such as anxiety and depression are common for people with muscular dystrophy, affecting most individuals in the community, according to a new survey. The survey was carried out by Bionews, the parent company of Muscular Dystrophy News. Survey results indicate that loneliness, regret, and…
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.
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