Duchenne

Mutations of the gene encoding dystrophins have long been known to cause the debilitating muscle-wasting disease DMD, which affects one in every 5,000 boys born. People with the condition will usually only live into their 20s or 30s. Now, a…

Researchers at Johns Hopkins Medicine report that an experimental drug first developed to treat kidney disease prolongs survival and improves muscle function in mice genetically engineered to develop a severe form of Duchenne muscular…

New research published in JAMA recommends daily steroid doses for children with Duchenne muscular dystrophy (DMD), marking a significant change in how the disease is treated. University of Rochester Medical Center (URMC) neurologist Robert…

A cell therapy developed by the executive director of the Smidt Heart Institute stabilizes weakened muscles-including the heart muscle-in Duchenne muscular dystrophy patients, a new study published in the international peer-reviewed journal…

One protein, Piezo1, is key to marshalling muscle stem cells' unique shapes and response to injuries, but it is in low supply in those with Duchenne muscular dystrophy, according to a team at the Perelman School of Medicine at the…

A clinical trial at UC Davis Health and six other sites showed that a cellular therapy offers promise for patients with late-stage Duchenne muscular dystrophy (DMD), a rare genetic disorder causing muscle loss and physical impairments in…

A new "cocktail" drug being developed at the University of Alberta could provide an effective and economical treatment to lessen symptoms for up to 45 per cent of patients with Duchenne muscular dystrophy (DMD), a chronic muscle-wasting…

Each year, about 20,000 children are diagnosed with Duchenne muscular dystrophy, a rare genetic condition that causes progressive muscle weakness and other systemic damage. Duchenne primarily affects males and is usually diagnosed by age 4.…