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 ...

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, ...

Gene therapies, exon-skipping drugs and next-gen steroids are reshaping Duchenne muscular dystrophy care, targeting broader ages and mutations. We are witnessing the fastest pivot in rare-disease ...

Melanie Sanford fought to get her son Hudson a breakthrough gene therapy to stop the progression of the fatal disease Duchenne Muscular Dystrophy. She rushed to get him access just days before he ...

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.