DMD is a devastating rare disease with a significant unmet need.
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births (about 20,000 new cases each year worldwide).
Because the Duchenne gene is found on the X-chromosome, it primarily affects boys; however, it occurs across all races and cultures.
Duchenne results in progressive loss of strength and is caused by a mutation in the gene that encodes for dystrophin.
Because dystrophin is absent, the muscle cells are easily damaged.
The progressive muscle weakness leads to serious medical problems, particularly issues relating to the heart and lungs.
Young men with Duchenne typically live into their late twenties. Duchenne can be passed from parent to child, but approximately 35% of cases occur because of a random spontaneous mutation.
In other words, it can affect anyone. Although there are medical treatments that may help slow its progression, there is currently no effective treatment or cure for Duchenne. (http://www.parentprojectmd.org).