Category: neurology

The US Food and Drug Administration has given the green light to Ionis Pharmaceuticals’ Zanvastro (zilganersen), the first disease-modifying drug for a rare, progressive and often fatal neurological condition known as Alexander disease. Zanvastro is an antisense oligonucleotide that targets RNA encoding glial fibrillary acidic protein (GFAP), which is overexpressed in astrocyte cells in Alexander disease because of mutations in the GFAP gene.

Zanvastro works by reducing GFAP overproduction in astrocytes. If there is too much GFAP, it forms clumps that cause astrocytes to malfunction and disrupt myelination in nerve-cell axons. People with Alexander disease have impaired motor function, seizures, intellectual disability and developmental delays; it affects about one person out of every 1–3 million.

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