Mycophenolate mofetil in myasthenia gravis: the unanswered question.

Myasthenia gravis (MG) is an autoimmune disease that leads to muscular weakness, which can significantly affect the patient's daily functions. If left untreated, the mortality rate can be as high as 30%. Effective immunosuppression is the cornerstone of treatment of MG, although most currently available immunomodulatory drugs are associated with unacceptable side effects, delayed onset of therapeutic action, or both. Mycophenolate mofetil (MMF) might be better tolerated than other immunosuppressants and many case reports and uncontrolled trials have indicated that it is effective in MG. However, two recently concluded clinical trials failed to demonstrate the efficacy of MMF in MG. This paper critically reviews the existing evidence on the efficacy of MMF in MG and provides the authors' view of its role in current practice.