Our lead program is designed to address the underlying genetic cause of Duchenne Muscular Dystrophy
~30,000 people in the U.S and Europe have Duchenne.
Duchenne Muscular Dystrophy – often referred to as DMD – is a rare disease caused by mutations in the DMD gene, which encodes for the dystrophin protein. These mutations lead to inadequate dystrophin production.
Dystrophin is essential to maintaining the structural integrity and function of muscle cells.
Lack of functional dystrophin leads to progressive loss of muscle strength, impacting mobility and causing heart or respiratory complications that contribute to high mortality rates.Duchenne causes progressive loss of muscle function throughout the body.To date, currently approved therapies for DMD have not shown any considerable increases in dystrophin production or any new skeletal muscle. In addition, none attack the final cause of death which is duchenne cardiomyopathy as we have two unique DMD drugs in the pipeline.
