Families living with Alexander disease have never had a treatment aimed at the disease itself. As of September 3, they do.
The Food and Drug Administration approved Zanvastro, known during development as zilganersen, for children and adults with Alexander disease, a rare and often fatal disorder that damages the brain's white matter. It affects roughly one in one to three million people worldwide, and fewer than 1,000 people in the United States are believed to have it. Until now, care meant managing seizures, feeding problems, and loss of movement as the disease advanced, with nothing to slow the underlying process.
