Tag: Alexander disease
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FDA Approves Ionis’ Zanvastro as the First Treatment for Alexander Disease
The FDA approved Ionis’ Zanvastro as the first treatment for Alexander disease, validating GFAP-targeted antisense therapy in an ultra-rare neurological disorder.
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How Zilganersen Targets Alexander Disease by Reducing GFAP RNA
Zilganersen is a GFAP-targeting antisense oligonucleotide that reduces GFAP production upstream of protein accumulation, astrocyte stress and white-matter injury in Alexander disease.