Less than half of patients had undergone bone monitoring, suggesting a need for better clinical guidance and management of ...

This leads to changes in joint and bone structure, which can affect organs such as the lungs and lead to an array of other ...

There are several types of muscular dystrophy. Muscle weakness is a hallmark of each type. But the symptoms can vary and start at different ages. Some muscular dystrophies are mild. Others are ...

Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...

Typically, a carrier of DMD will not show symptoms. However, some individuals may show mild symptoms, such as muscle weakness. Likelihood of passing on the DMD gene variation The chance of passing ...

Enrollment of healthy volunteers for a Phase 1 trial testing Duchenne muscular dystrophy (DMD) treatment candidate SAT-3247 ...

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from ...

The company is evaluating whether the drug, which targets cardiac complications, might be used treat patients with other ...

While current treatments can help manage some DMD symptoms, there are no approved therapies specifically targeting DMD-related heart disease, highlighting a critical unmet medical need.