Duchenne Muscular Dystrophy
DMD is a genetic disorder characterized by the progressive degeneration and loss of skeletal muscle, the muscles of respiration and the cardiac muscle, primarily in boys. DMD is the most prevalent form of human muscular dystrophy. Prevalence ranges from 1:3000 to 1:3500 boys.
DMD is caused by mutations of the DMD gene on the short arm(p) of the X chromosome. The gene regulates the production of dystrophin, a 427 kDa cytoskeletal protein, that is found in association with the membrane of skeletal and cardiac muscle cells.
Dystrophin is thought to play an important role in maintaining the structure of these muscle cells. DMD patients are usually wheelchair-bound by 12 years of age and die of respiratory failure in their late teens or early twenties.
A significant global research effort is underway to identify drugs and novel advances in the care of DMD patients by delaying the progression of DMD-related disability and prolonging survival.
Administration of glucocorticoids is among these advances in reducing inflammatory processes in muscles of boys with DMD suggesting that immune cell regulation may become an effective option in treating individuals with Duchenne.
Glucocorticoid therapy, however, is associated with diverse potential adverse effect.
Our approach is different. SymThera's approach is to slow the progress of the disease by regulating the large contribution of immune cell responses to the death of dystrophic muscles.
We target proteins that are derived from three major categories: muscle proteins, inflammatory proteins and extracellular matrix (ECM) proteins to reduce inflammation, and improve muscle function.
We believe short sequence peptides derived from the three major categories of proteins will impact the DMD-associated proteins network.