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  • Ionis’ antisense drug stabilizes ultra-rare disease ahead of FDA decision
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Ionis’ antisense drug stabilizes ultra-rare disease ahead of FDA decision

BioSpace

The pivotal study of zilganersen in Alexander disease missed a secondary endpoint, but analysts expect the FDA to approve the asset given the unmet need and overall data.

Clinical DataRare DiseaseRead full story

pharminent April 22, 2026 (Last updated: April 22, 2026)

BioSpace

The pivotal study of zilganersen in Alexander disease missed a secondary endpoint, but analysts expect the FDA to approve the asset given the unmet need and overall data.

Clinical DataRare DiseaseRead full story

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