MDA criticized a DOJ memorandum on disability care, saying it threatens in-home and community-based services for people with ...

Being part of the PPMD conference's Adult Advisory Committee has helped columnist Patrick Moeschen bond with those who share ...

Health Canada is granting priority review to givinostat, sold as Duvyzat in the U.S., as an oral treatment for Duchenne ...

A CITGO golf fundraiser in Louisiana raised a record $756,800 for MDA to support people living with neuromuscular diseases ...

Muscular dystrophy (MD) refers to a group of inherited muscle disorders caused by mutations in genes that generate proteins that play an essential role in muscle structure and function. The disease ...

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.

People with Duchenne muscular dystrophy (DMD) require some level of caregiving throughout their whole lives, particularly as the disease progresses and patients become less mobile and more reliant on ...

While Duchenne muscular dystrophy (DMD) is one of the most common types of this group of muscle-wasting disorders, affecting about 1 in 3,500 male births, each patient’s journey with this form of ...

Going to school for the first time is an important moment in a child’s life. The educational journey for a child with Duchenne muscular dystrophy (DMD) can be exciting but also challenging. To smooth ...

Bridgebio Pharma has submitted an application asking the U.S. Food and Drug Administration (FDA) to approve its experimental oral therapy, BBP-418, to treat limb-girdle muscular dystrophy type 2i ...

For parents and caregivers, a new diagnosis of Duchenne muscular dystrophy (DMD) can feel overwhelming. There’s no road map to managing the physical and mental changes in your child’s health, ...

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 ...