LHON-MS patient with long-term non-response to Idebenone shows improvement after 4-AP Usage

Leber's hereditary optic neuropathy (LHON) is a maternally inherited mitochondrial disorder with a poor visual prognosis due to the loss of retinal ganglion cells and degeneration of the optic nerve head. There are 3 primary mutations: m.11778A>G, m.3460A>G, and m.14484T>C(ordered from worst to best prognosis). VA is primarily affected with a concordant decrease in central vision. Multiple sclerosis is a multisystem immune-mediated process in which an abnormal response of the body's immune system is directed toward the central nervous system. Although there is no direct connection between LHON and MS, if they cooccur there may be utility in the use of adjunctive therapy.