The U.S. Food and Drug Administration (FDA) has allowed the enrollment of up to 30 additional boys with Duchenne muscular dystrophy (DMD) in a clinical study testing an experimental stem cell therapy. The Phase 2 trial (NCT06579352) testing the treatment from Signature Biologics has enrolled five boys,…
Discussion
My family recently spent a week in Indiana with friends at a cabin on a lake, and it was so good for us. Our two families met more than a decade ago when our sons were all little boys, and we have remained friends throughout our separate journeys with…
Discussion
A U.S. biopharmaceutical company is gearing up to launch its first clinical trial of a novel cell therapy designed to restore lost muscle cells in people with Duchenne muscular dystrophy (DMD). Texas-based IPS Heart announced that it is preparing an investigational new drug application or IND — a formal…
Discussion
My regular readers know I’ve been losing function in my right arm, making many activities of daily living challenging. The pain, weakness, and stiffness I experience when I try to do anything with that arm began suddenly and has progressed rapidly this year. I’m now experiencing similar symptoms in my…
Discussion
When muscle fibers fail to properly seal tiny tears in their membrane, as occurs in some forms of muscular dystrophy, the resulting leakage can change the surrounding environment and attract immune cells that contribute to further muscle damage, a mouse study suggests. The findings suggest that the tissue surrounding…
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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