Rare Diseases: Neuromyelitis Optica


Neuromyelitis Optica (NMO) is a rare autoimmune disease that stems from immune reactivity to self-antigens, primarily Aquaporin-4, found on cells in the eye and the central nervous system. NMO is characterized by symptoms such as blindness, weakness or paralysis, loss of sensation, and spinal cord damage.

Existing therapies in use in the clinic or under development for the treatment of NMO are aimed at broadly suppressing the immune system. While these therapies relieve symptoms, they do not address the underlying causes driving disease and result in severe side-effects.