About

Alexander disease is a rare neurological disorder where faulty myelin production damages nerve fibers in the brain, causing developmental delays, seizures, and progressive loss of motor control in children. Zilganersen is an experimental drug injected into the spinal fluid that blocks the genetic instructions for making GFAP, the abnormal protein driving this damage. By reducing GFAP production at its source, the treatment could potentially slow or stabilize the disease's progression in affected patients.