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…
The latest Phase 3 trial results continue showing that deramiocel, an investigational cell therapy for heart disease related to Duchenne muscular dystrophy (DMD), significantly slows the progression of arm and heart damage in boys and men with DMD. Based on the positive Phase 3 results, Capricor Therapeutics has…
Myotonic dystrophy type 1 (DM1) patients in a Phase 1/2 clinical trial saw gains in motor function and cognitive measures after receiving Dyne Therapeutics’ zeleciment basivarsen (z-basivarsen), previously known as DYNE-101, supporting the recent initiation of a Phase 3 study. Findings from the Phase 1/2 ACHIEVE trial…
Treatment with Duvyzat (givinostat) was associated with functional gains in boys with Duchenne muscular dystrophy (DMD) regardless of the final dose they received, according to new analyses from a Phase 3 clinical trial. The treatment also led to a slower loss of functional muscle tissue and a slower replacement…
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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