FDA approves first therapy for Alexander disease - Takeaways - MDSpire

FDA approves first therapy for Alexander disease

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

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The FDA approved zilganersen (Zanvastro) for treating Alexander disease in pediatric and adult patients.
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Alexander disease is a rare neurologic condition caused by mutations affecting glial fibrillary acidic protein production.
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Zilganersen is an antisense oligonucleotide that reduces abnormal GFAP accumulation in the brain.
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Clinical trials showed improved walking speed in patients aged 5 years and older receiving zilganersen.
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Common adverse reactions to zilganersen include vomiting, back pain, cough, headache, and aseptic meningitis.