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Evidence for efficacy of a drug widely used without authorisation in multiple sclerosis: mycophenolate mofetil
  1. Jun-ichi Kira
  1. Correspondence to Professor J-I Kira, Department of Neurology, Neurological Institute, Graduate School of Medical Sciences, Kyushu University, Fukuoka 812-8582, Japan; kira{at}neuro.med.kyushu-u.ac.jp

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Mycophenolate mofetil (MMF) is an oral DNA base synthesis inhibitor that has profound immunosuppressive effects on activated T cells, B cells and macrophages. MMF selectively inhibits inosine 5′-monophosphate dehydrogenase type II, responsible for de novo synthesis of the purine nucleotide guanine. The drug has been used for anti-rejection therapy in organ transplantation and also for immunotherapy in a myriad of autoimmune diseases, including refractory multiple sclerosis (MS). The efficacy of MMF has been examined in several preliminary prospective studies using relatively small numbers of active relapsing–remitting MS patients, in which MMF was introduced as an add on therapy for interferon β.15 These studies mostly showed …

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