alexander disease

1 entry tagged alexander disease. All writing.

SEPTEMBER 21, 2026 Alexander Disease, and the First Drug Approved to Treat It What is Alexander disease, and what did the FDA actually approve for it? AxD is an ultra-rare, usually fatal genetic disorder of the brain's astrocytes, caused by mutations in the GFAP gene. On September 3, 2026, the FDA approved Zanvastro (zilganersen), an RNA-targeted drug given by quarterly spinal injection, as the first treatment aimed at the disease itself rather than its symptoms. The trial behind it enrolled 49 randomized patients.

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