A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

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Duchenne muscular dystrophy (DMD) is a genetic disease ... DNA testing, and a muscle biopsy. Blood testing can detect elevated creatine kinase (CK) levels in your child’s blood.

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, ...

WVE-N531 showed significant improvements in functional measures and muscle health in DMD patients during the phase 2 FORWARD-53 trial. Sustained exon skipping, dystrophin restoration, and improved ...

Suneel Ram's caregivers share some of the lessons they have learned while caring for the 28-year-old who was diagnosed with ...

DMD was first described by French neurologist Guillaume Benjamin Amand Duchenne in the 1860s. However, until the 1980s, ...

Becker muscular dystrophy (BMD) is a type of muscular dystrophy, a genetic condition that weakens and damages muscles. It can worsen with age. However, its symptoms are less severe than those of ...

Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular ...

Duchenne muscular dystrophy is characterized by a mutation in the DMD gene that leads to a lack of dystrophin and muscle loss. It affects about one in 3,300 boys. Delandistrogene moxeparvovec ...

Duchenne muscular dystrophy is a condition that primarily affects young boys, starving their bodies of dystrophin protein required for muscle function. The Food and Drug Administration has said ...