About

Alexander disease is a rare neurological disorder that damages the protective myelin around nerve fibers in the brain, typically causing developmental delays, loss of motor control, and seizures in children. Zilganersen is an antisense oligonucleotide designed to be delivered directly into the spinal fluid to reduce the production of the GFAP protein. By targeting this protein at its genetic source, the drug may help stabilize or improve motor function in affected individuals.