Families living with Alexander disease have had one option for as long as the condition has been recognized, which is to manage symptoms and watch the disease advance. That changed on September 3.
The Food and Drug Administration approved Zanvastro, known generically as zilganersen, for the treatment of Alexander disease in children and adults. It is the first approved therapy for the condition and the first designed to act on the protein buildup that drives it rather than the symptoms it produces.