As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are caught in the middle.

Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles.

The CHMP recommends conditional marketing authorization for Duvyzat to treat Duchenne muscular dystrophy in patients aged 6 years and older who can walk.

Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

Suneel Ram is a spiritual seeker, sings in a band and was the subject of a comic novel two years ago about living with Duchenne muscular dystrophy, a rare genetic disorder that afflicts one in ...

The Cambridge-based biotech's experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the stage for a new therapeutic option for patients with the rare ...

Duchenne is still not part of the RUSP though certain states have taken initiatives to add it to their newborn screening panels with advocacy efforts by Parent Project Muscular Dystrophy and others.

The global Duchenne muscular dystrophy drugs market size reached USD 2.5 Billion in 2024. Looking forward, the market is projected to reach USD 5.9 Billion by 2033, exhibiting a growth rate (CAGR ...

The FDA expanded the approval of delandistrogene moxeparvovec-rokl (Elevidys) gene therapy for Duchenne muscular dystrophy on Thursday to include ambulatory or non-ambulatory patients ages 4 years ...

My son's smile gives me hope for Duchenne muscular dystrophy. A new law could help others Opinion: Newborn testing can give families a head start on giving a child with the degenerative muscular ...

Positive results observed in Duchenne muscular dystrophy patients undergoing gene therapy mark a breakthrough in Illinois medical treatment.

The Minnesota Department of Health on Friday announced that it will add Duchenne muscular dystrophy to the list of conditions for which Minnesota newborns are typically screened.