What is Duchenne Muscular Dystrophy?

Muscular dystrophy is a rare genetic condition. There are nine different types of muscular dystrophy total. Duchenne Muscular Dystrophy refers to a form of the condition where the patient does not produce enough dystrophin within their muscles. Worldwide, DMD affects approximately one in every 3,500 to 5,000 males. It is much less common in females.

The condition results in muscle weakness, fatigue, and difficulty with motor skills among other symptoms. Eventually, it affects the heart and respiratory system. By the age of 15, more than 90% of individuals with DMD are confined to a wheelchair.

The Trial

The trial being conducted by Capricor Therapeutics is called “HOPE-2”. It is investigating CAP-1002 as a potential treatment for DMD. However, it was placed on hold due to a severe allergic reaction which occurred after the drug was administered to a patient. The patient was treated and thankfully is now doing well.

Fortunately, this allergic reaction has not caused Capricor to cancel HOPE-2, the company is simply putting things on pause to consult with the FDA. Together, they are coming up with a mitigation plan.

Hopefully, we will see this trial resumed shortly. In the meantime, you can read more about HOPE-2 and the event which caused the hold here.