After 30 years, scientists finally have a drug for a deadly brain disease

The FDA has approved Zanvastro, the first drug designed to directly treat Alexander disease, a rare neurological disorder affecting fewer than 1 in a million people. After three decades of research, clinical trials showed the treatment stabilized motor function and improved movement in some patients.

Zanvastro has completed clinical trials; it is the first disease-modifying treatment for Alexander disease, which has no previous approved medications targeting the underlying condition.