FDA approves first disease-modifying therapy for Alexander disease
The FDA approved Ionis Pharmaceuticals' drug Zanvastro as the first disease-modifying therapy for Alexander disease, a rare and deadly neurological disorder previously without approved treatment. The approval applies in the United States. Long-term real-world outcomes are not yet known.
Why it matters
This approval provides the first targeted treatment for a fatal neurological condition that previously had no disease-modifying options.
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