Aim:
Use drugs to treat the various aspects of disease pathology. While these treatments do not restore dystrophin production, addressing the pathological consequences of dystrophin loss can slow down disease progression.

Background:
Due to the loss of dystrophin, patient’s muscle fibers are continuously damaged during exercise. Lost muscle tissue is replaced by scar tissue (‘fibrosis’) and fat tissue (“adiposis”). This process is irreversible and is exacerbated by an immune response that is initiated by the muscle damage. Drugs can help to increase muscle growth, to compensate for the lost muscle tissue. Alternatively, they can suppress the immune system or inhibit scar tissue formation.

There is a vast number of drugs that are reputedly beneficial in Duchenne patients and/or dystrophic mouse models. In this section we list the ones that have been tested in patients and the ones that gave very promising results in mouse models.