Center for Duchenne Muscular Dystrophy

The Center for Duchenne Muscular Dystrophy is the first of its kind in the west and a cornerstone of the new California Center for Rare Diseases at UCLA. The Center is a unique collaboration between UCLA scientists and clinicians who realized the need for an integrated multidisciplinary center to care for children and young adults suffering from muscular dystrophy, a debilitating rare genetic disease that delivers a progressive loss of muscle movement and a life expectancy of 25 years. The Center’s efforts span education and outreach, leading-edge research, drug discovery, clinical trials and care. Scientists at UCLA developed Exondys 51, the first FDA-approved therapeutic drug for the