A large clinical trial evaluating the treatment candidate sevasemten for people with Becker muscular dystrophy (BMD) has failed to meet its main goal, with “no statistically significant benefit” seen on a measure of physical function. That’s according to top-line data, or the first study results, from GRAND…
Discussion
My first caregivers were my parents. This is true for the vast majority of us. If everyone is fortunate to live long enough, the roles often flip, with grown children caring for their aging parents. But when you factor in muscular dystrophy, this model no longer holds. Growing up,…
Discussion
“Take care of yourself.” I hear these four little words so often, yet they can be hard to follow. I have seven children with my husband, Jason: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 12; and Callie, 4. Max, Rowen, and Charlie have Duchenne muscular…
Discussion
Treatment with the experimental gene therapy BB-301 was found to improve swallowing ability for every person with a rare type of muscular dystrophy (MD) that mainly affects the throat and eyes who are taking part in a first-in-human clinical trial. That’s according to new interim data announced by Benitec…
Discussion
I have an ambitious bucket list for someone who needs help with almost everything physically. I want to watch Chelsea play at Stamford Bridge, their home stadium in West London, preferably on a Champions League night. I’d love to write my memoir and pursue a master’s degree in economics, systems…
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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