Yes. In September 2026 the U.S. Food and Drug Administration approved Zanvastro, the first and only disease-modifying treatment for Alexander disease — a rare, progressive neurological disorder that can impair motor, cognitive, and autonomic function. Until this approval, care was limited to managing symptoms.
The drug grew out of more than 30 years of research at the University of Wisconsin–Madison (with Ionis Pharmaceuticals) targeting excess glial fibrillary acidic protein (GFAP). Zanvastro is an antisense oligonucleotide given by injection into the spinal canal every three months; in a 54-patient trial, people treated for a little over a year had significantly better walking speeds than untreated patients, and the FDA cleared it for infancy through adulthood.
Source: UW–Madison News — First drug to treat Alexander disease approved by the FDA