What is Alexander disease—and how does the first-ever drug for it work?

Zanvastro is the first approved treatment aimed at the GFAP buildup that drives Alexander disease.

1. Alexander disease is a rare progressive neurological disorder linked to abnormal GFAP accumulation.
2. Zanvastro is an antisense medicine designed to reduce production of the harmful protein.
3. A trained professional injects it into the spinal canal every three months; it is not a daily pill.
4. Patients and caregivers should discuss diagnosis, monitoring, trial evidence, availability, and individual risks with a specialist.