Zilganersen, Alexander Disease, Rare Disease, Neurology, FDA PDUFA

Zilganersen, Alexander Disease, Rare Disease, Neurology, FDA PDUFA

Zilganersen ASO Reverses Functional Decline in Alexander Disease Ahead of 2026 PDUFA

First-in-class antisense oligonucleotide demonstrates age-dependent functional improvements and robust biomarker reduction, overcoming the absolute limitations of palliative standard of care. See Disclaimer below * Alexander disease is an ultra-rare leukodystrophy that has, until now, sat entirely outside the reach of disease-modifying therapy. Global prevalence sits between 1 in 1,000,000