5 Key Takeaways

• 1

  The FDA approved zilganersen (Zanvastro) for treating Alexander disease in pediatric and adult patients.

• 2

  Alexander disease is a rare neurologic condition caused by mutations affecting glial fibrillary acidic protein production.

• 3

  Zilganersen is an antisense oligonucleotide that reduces abnormal GFAP accumulation in the brain.

• 4

  Clinical trials showed improved walking speed in patients aged 5 years and older receiving zilganersen.

• 5

  Common adverse reactions to zilganersen include vomiting, back pain, cough, headache, and aseptic meningitis.