Abstract
Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune inflammatory demyelinating disorder of the central nervous system (CNS), which is a severely disabling disorder leading to devastating sequelae or even death. Repeated acute attacks and the presence of aquaporin-4 immunoglobulin G (AQP4-IgG) antibody are the typical characteristics of NMOSD. Recently, the phase III trials of the newly developed biologicals therapies have shown their effectiveness and good tolerance to a certain extent when compared with the traditional therapy with the first- and second-line drugs. However, there is still a lack of large sample, double-blind, randomized, clinical studies to confirm their efficacy, safety, and tolerability. Especially, these drugs have no clear effect on NMOSD patients without AQP4-IgG and refractory patients. Therefore, it is of strong demand to further conduct large sample, double-blind, randomized, clinical trials, and novel therapeutic possibilities in NMOSD are discussed briefly here.