The comment comes in many forms: “I don’t know how you do it.” “You have your hands full!” “I can’t imagine what it would be like.” It’s always in response to seeing my large family. I share seven children with my husband, Jason: Lexi, 24; Max, 20; Chance, 18; Rowen,…
Dyne Therapeutics is advancing its investigational exon-skipping therapy zeleciment rostudirsen (z-rostudirsen), formerly known as DYNE-251, toward regulatory approval after trial data showed early signs of benefit across multiple disease measures in boys with Duchenne muscular dystrophy (DMD). The most recent data from the Phase 1/2 DELIVER clinical trial…
In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To…
Boys with Duchenne muscular dystrophy (DMD) who received Sarepta Therapeutics’ gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in a clinical trial continue to show signs of slowed disease progression relative to DMD’s natural course, along with caregiver-reported improvements in everyday living. That’s according to data from the Phase…
Discussion
For most of my life, Duchenne muscular dystrophy has defined how I navigate the world. Duchenne is a genetic condition that causes progressive muscle loss. Over time, it weakens the arms, legs, breathing muscles, and heart. Many adults with Duchenne, including me, use wheelchairs and rely on a…
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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