Sarepta Therapeutics (SRPT) announces that the U.S. Food and Drug Administration has accepted the Company’s Biologics License Application seeking accelerated approval of SRP-9001 for the treatment of ambulant individuals with Duchenne muscular dystrophy
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Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and is eventually fatal. The genetic change that causes Duchenne—a mutation in the DMD gene—happens before birth and can be inherited, or new mutations in the gene can occur spontaneously. Muscle weakness becomes increasingly noticeable between the ages of 3 and 5, and most patients use a wheelchair by the time they are 12. During adolescence, heart and breathing muscles weaken, leading to serious, life-threatening complications.
Duchenne affects approximately 1 in 3,500 to 5,000 males born worldwide.