title: [The treatment of mitochondrial disorders, present and future].
abstract: Mitochondrial disorder has an inherited multi-system mitochondrial dysfunction that often involves the nervous, endocrine, renal and cardiac system. Although many molecular and cellular mechanisms leading to mitochondrial cytopathy have been discovered, clinical management of the disorders remains largely supportive. Many therapeutic drugs and reagents have been published effective in the past 20 years, however, none of those have been approved their efficacy by a double blind randomized placebo-controlled, or open labeled trial. In this review, we describe the current situation for developing the therapeutic drugs in mitochondrial disorders by clinical trial registry. We also describe the investigator-mediated clinical trial of L-arginine, and taurin for MELAS, and new project for developing the therapeutic drug of sodium pyruvate for lactic acidosis associated with mitochondrial disorders in Japan.
authors.affiliation: Department of Pediatrics and Child Health, Kurume University School of Medicine.
authors.forename: Yasutoshi
authors.initials: Y
authors.lastname: Koga
mesh_terms: D006801:Humans; D017241:MELAS Syndrome / Q000188:drug therapy*