Refractory Myasthenia Gravis – Patient Burden and the Need for New Therapeutic Targets

Abstract

Myasthenia gravis (MG) is an autoimmune disorder characterised by muscle weakness and fatigue. Symptoms include slurred speech, weakness in the arms and legs and difficulty swallowing and breathing, which can lead to life-threatening myasthenic crisis. Despite receiving recommended therapies, many patients with MG report health-related quality of life is low, largely due to impaired mobility and depression. Furthermore, around 10% of patients with generalised MG fail to respond to treatment. Patients with refractory MG have a severely compromised quality of life and it is important that new treatments are evaluated for this group of patients.