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With current corticosteroid use, people with Duchenne muscular dystrophy (DMD) typically lose their walking ability by their early teens, often need ventilation by their late teens, and usually survive until the third or fourth decade of life, according to a review study into the clinical course of DMD today.
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High levels of the toxic RNA molecule associated with myotonic dystrophy type 1 (DM1) lead to defects in muscle regeneration and in the number of muscle stem cells — or satellite cells — and promotes muscle fibrosis (scarring) upon injury, according to a study in a mouse model of DM1.
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000 in grants, totaling…
Top-line data from the VISION-DMD Phase 2b clinical trial evaluating vamorolone in boys with Duchenne muscular dystrophy (DMD), showed efficacy across primary and secondary study goals. The investigational therapy was also well-tolerated with a…
When her son initially had a feeding tube placed, columnist Prudence Bloom was concerned. Now, she realizes it’s a blessing.
Visit the Muscular Dystrophy News Today forums to connect with others in the muscular dystrophy community.
The Muscular Dystrophy News Today team provided in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference in March. Check out all the latest news from the meeting by clicking the link below.
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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