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

A long-term study found that heart health is generally stable over time in women carrying mutations that can cause MD types ...

Your clinical hub for comprehensive DMD management. Explore expert-reviewed resources focused on early diagnosis, optimizing multidisciplinary interventions, and navigating emerging genetic therapies ...

Assistive technology refers to any computer-controlled product, gadget, device, application, or service that assists, maintains, or improves the abilities of individuals with motor disabilities. The ...

A human being since 1972, Patrick Moeschen recently retired after 28 years of teaching music at the middle school level in a public school setting. Diagnosed with Becker muscular dystrophy (MD) in ...

Exon skipping is a treatment approach for people whose Duchenne muscular dystrophy is due to certain mutations in its causative gene. Exon skipping works like a molecular patch, so that the DMD gene ...

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 ...

“Take him home and give him a good life.” This was the main advice given to Jessica and Chris Curran by a genetic counselor after their son, Conner, was diagnosed with Duchenne muscular dystrophy (DMD ...

Muscular dystrophy entered my life in the fall of 1984. I was 11 years old and beginning my sixth year of school. Along with neighborhood friends, I would walk to and from my elementary school every ...

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 ...

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.

Dyne Therapeutics is advancing its investigational exon-skipping therapy zeleciment rostudirsen (z-rostudirsen), formerly known as DYNE-251, toward regulatory approval after trial data showed early ...