"High clinical suspicion is important for this population of patients. Since muscular dystrophy is progressive in nature, monitoring disease progression in these patients is critical for timing ...

With improved survival becoming more common among individuals who have Duchenne muscular dystrophy (DMD), cardiomyopathy is also increasing in prevalence among this population. New data from a 20-year ...

Boys with Duchenne muscular dystrophy (DMD) have a clinically proven, new treatment option with the Food and Drug Administration's approval Thursday of vamorolone, a steroidal-type, anti-inflammatory ...

While it’s established that Duchenne muscular dystrophy (DMD) causes a loss of muscle mass and strength, few studies have examined the gastrointestinal issues tied to DMD. Initial fractures are the ...

SGT-003 continues to be generally well tolerated with 36 participants dosed as of February 9, 2026, in Phase 1/2 trial - ...

Muscular dystrophy is a group of diseases that make muscles weaker and less flexible over time. It is caused by a problem in the genes that control how the body keeps muscles healthy. Duchenne ...

With the writing apparently on the wall, PTC Therapeutics has called off its latest bid for FDA approval of its Duchenne muscular dystrophy (DMD) drug Translarna.  | Late Thursday, PTC revealed that ...

Blocking a certain type of ion channel has been shown to prolong survival in mouse models of severe Duchenne muscular dystrophy (DMD), which could lead to the development of novel treatments in humans ...

Your muscles need continuous maintenance to stay strong and healthy. Duchenne muscular dystrophy (DMD) happens when there is a change in a person's genetic instructions that affects the production of ...

CORAL GABLES, Fla. — There’s new hope for children born with a fatal form of muscular dystrophy. Nicklaus Children’s Hospital is one of the first in the nation and one of only three hospitals in ...

For genetic conditions like Duchenne muscular dystrophy, there is little doctors can do to slow or treat the condition other than trying to manage symptoms, since only addressing the genetic changes ...

The degree to which novel value elements such as insurance value impact estimated treatment value for rare, severe genetic diseases such as Duchenne muscular dystrophy is unclear. Objectives: To ...