Dr Essajee says that in Kenya, treatment is largely supportive: “Steroids like Deflazacort, combined with vitamin D and ...

University of Manchester scientists have mapped the mutations in the tiny protein chains that cause a subtype of muscular ...

Agamree, an approved steroid treatment for Duchenne MD, will be sold in Turkey by GEN, per an agreement with Santhera.

I n the next few weeks, Sarepta Therapeutics will release new data that could change the future of the company and the lives of thousands of patients with Duchenne muscular dystrophy. The Phase 3 ...

University of Manchester scientists have mapped the mutations in the tiny protein chains that cause a subtype of muscular ...

Managing osteoporosis and bone health in Duchenne muscular dystrophy occurs in a high-risk environment for fractures due to disease-related loss of muscle and movement, adverse effects of ...

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a favorable safety profile in young patients. Delandistrogene moxeparvovec ...

Muscular Dystrophy is a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing them to weaken and deteriorate over time. The most common form of muscular ...

Charlie has Duchenne muscular dystrophy, a degenerative genetic disease that, until recently, has guaranteed death by early adulthood from cardiac or respiratory failure.

Cardiomyopathy is the leading cause of death in patients with Duchenne muscular dystrophy, with roughly 90% developing a dilated cardiomyopathy by age 18. 2,3 Cardiomyocyte degeneration results in ...