Capricor receives rare pediatric disease designation for DMD drug | DuchenneXchange

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Capricor receives rare pediatric disease designation for DMD drug


The U.S. Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation to CAP-1002 to treat Duchenne muscular dystrophy (DMD).

If approved, CAP-1002 manufacturer, Capricor Therapeutics would receive a Priority Review Voucher which can be worth millions of dollars.

DMD is a rare genetic disorder that results in progressive degeneration and muscle due to disruption of the dystrophin protein complex in muscle tissue. DMD patients typically lose the ability to walk between the ages of 10 to 12 and typically die in their 20’s of heart failure. There approximately 15,000 to 20,000 affected people in the U.S

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