Targeting myostatin for therapies against muscle-wasting disorders.

In addition to gene correction therapy and cell transplantation techniques, multidisciplinary approaches to drug discovery and development offer promising therapeutic strategies for intractable genetic muscular disorders including muscular dystrophy. Inhibition of the production and activity of myostatin, a potent growth factor that determines skeletal muscle size, is a novel strategy for the treatment of muscle-wasting disorders such as muscular dystrophy, cachexia and sarcopenia. Myostatin blockers include myostatin-blocking antibodies, myostatin propeptide, follistatin and follistatin-related proteins, soluble myostatin receptors, small interfering RNA and small chemical inhibitors. This review describes the discovery and development of myostatin inhibitors.