Clinical features of an adult-onset Leigh syndrome caused by the T9176C mutation in the mitochondrial DNA ATPase 6 gene

Several lines of evidence support early immunomodulatory treatment of relapsing multiple sclerosis with either recombinant beta-interferons or glatiramer acetate and positive results from phase III trials encourage start of treatment even in patients with clinical isolated syndrome (CIS). However, currently available drugs for basic therapy are only partially effective and patients may still encounter relapses or disease progression. As treatment-refractory, clinically active MS can quickly lead to irreversible neurological disability there is an urgent need for more effective therapeutic strategies. The major goal is to reduce the damage, protect the tissue and enhance repair. In order to achieve these goals we not only need effective and safe drugs but also the appropriate study designs to really detect the most relevant outcomes 2, 7.