Duchenne Muscular Dystrophy: A Not-So-Rare Disease

Duchenne muscular dystrophy (DMD) is a rare and devastating muscle disease caused by mutations in the X-linked dystrophin gene, DMD. Since the first identification of the disease in the 1800’s, the clinical course of this complex disease has not changed; however, clinicians, scientists, and patients worked to improve the natural history of these patients. Together, we still continue to learn more about the patient and the disease. Novel therapies are continuously being investigated.

One of the biggest gaps for testing new therapies for DMD and other diseases is the lack of verifiable biomarkers. Serum biomarkers for DMD and other diseases hold significant potential as objective phenotypic measures of disease state, deeper insights to disease pathogenesis, new therapeutic approaches, and responses to therapeutic interventions. Clinicians and scientists continue to learn from the history of DMD and past therapeutic approaches. The identification and use of verifiable biomarkers will provide a rich body of valuable information in the search for new diagnostic, prognostic, and therapeutic approaches for patients with DMD and other diseases.