As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we ...

Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...

At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...

The company had previously halted shipments to certain older patients following the deaths of two teenagers treated with the ...

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscular weakness and mild intellectual disability.

More than 30 types of muscular dystrophy have been identified. They all cause problems with movement due to genetic mutations.

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD). WVE ...

A Fix for Muscle Mutations The colorful threads in this image are muscle fibers. This spotted rainbow illustrates a potential gene therapy approach to treating Duchenne muscular dystrophy (DMD) – a ...

Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

A single-center study of 555 individuals with Duchenne muscular dystrophy (DMD) treated with corticosteroids revealed that certain genetic mutations are linked to either delaying or accelerating ...

How Is Duchenne Muscular Dystrophy Diagnosed? DMD is diagnosed through a physical evaluation and blood tests to identify mutations in the DMD gene. Muscle samples might also be evaluated.