Leave a little sharper than you arrived.

FDA greenlights first-ever disease-modifying treatment for Alexander disease

After decades of supportive-care-only management, Alexander disease patients finally have a real treatment option. The FDA has approved Zanvastro (zilganersen), an antisense oligonucleotide that targets the toxic GFAP protein buildup driving this ultra-rare, often fatal neurological disorder. The approval covers pediatric and adult patients — from infancy through adulthood — marking a landmark moment for a community of roughly 300 people in the U.S.