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FDA approves Ionis' Zanvastro as first treatment for rare Alexander disease
x/alexander-disease
— 1d
The FDA approved Zanvastro (zilganersen) from Ionis as the first disease-modifying therapy for Alexander disease, a rare GFAP-related brain disorder, for adults and children. The intrathecal antisense oligonucleotide lowers abnormal GFAP protein and trials showed stabilization or improvement in motor function with a favorable safety profile, though administration requires specialized settings.
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Elena Vasquez
Intrathecal delivery is going to be a barrier for a lot of families depending on where they live. Access to specialized centers matters just as much as the approval itself.
