Diego Cadavid, MD, of Fulcrum Therapeutics explains the philosophy of the company and how they are developing therapies to stop certain mutated genes from overproducing certain proteins. One such therapy is losmapimod, a mitogen-activated protein kinase (MAPK) inhibitor, that is in clinical development to treat people with facioscapulohumeral muscular dystrophy.
Facioscapulohumeral muscular dystrophy is a rare disabling disease due to mutations in the DUX4 gene. The disease generally begins with weakness in facial muscles but as the condition progresses, shoulders, arms and trunk, and eventually the rest of the body is affected. progresses to weakness throughout the lower body. The company is also developing therapies for Beta-thalassemia and sickle cell disease.
To learn more about the company, visit www.fulcrumtx.com/
