The FDA updated prescribing information for Elevidys with new safety warnings and limiting its use to DMD patients who can ...

Agamree is as effective as traditional corticosteroids at preserving walking ability but carries a lower risk of side effects ...

Sarepta said DMD treatments Amondys 45 and Vyondys 53 show 'positive and encouraging trends' even though a trial failed to ...

Columnist Robin Stemple and others in the Western Pennsylvania chapter of the FSHD Society raised $85,000 for research toward ...

Dosing has begun in a long-term extension study testing Satellos Bioscience’s experimental oral therapy SAT-3247 in men with DMD.

In an effort to improve his heart health, columnist Shalom Lim trials a new medication for his cardiomyopathy, a complication ...

Histone deacetylase inhibitors are a treatment approach designed to work by blocking an enzyme that turns off gene activity. This may ultimately help by slowing muscle loss and disease progression.

Exon skipping is a treatment approach for people with Duchenne muscular dystrophy (DMD) that, while not a cure, may help to make the effects of the genetic disease less severe. Duchenne, the most ...

Caring for three sons with Duchenne MD means that all columnist Betty Vertin has left are the leftovers, but this week, even they ran out.

Betty Vertin is a mother and writer living in rural Hastings, Nebraska, with her husband and seven children. Betty is a caregiver to three sons, Max, Rowen, and Charlie. Her oldest was diagnosed with ...

A young man with Duchenne muscular dystrophy (DMD) who received the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) has died due to acute liver failure. In a statement, the ...

Muscular dystrophy refers to a group of genetic diseases that cause progressive muscle weakness and loss. There are more than 30 types of muscular dystrophy, all with different causes and symptoms.