FDA approves first therapy for Alexander disease - Takeaways - MDSpire
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FDA approves first therapy for Alexander disease

The antisense oligonucleotide reduces production of the abnormal protein that accumulates in supportive cells in the brain.

  • By

  • Kathryn Wighton

  • September 4, 2026

  • 2 min

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  • 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.

Original Source(s)

FDA approves first therapy for Alexander disease

  • by Kathryn Wighton

  • September 4, 2026

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