For more than two decades, molecules that block the myostatin signaling pathway have been investigated in a broad range of muscle diseases, including facioscapulohumeral muscular dystrophy, Becker muscular dystrophy, limb-girdle muscular dystrophy, Duchenne muscular dystrophy (DMD) and inclusion body myositis. The gene discussed is MSTN; the disease is Duchenne muscular dystrophy.