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Ionis Pharmaceuticals Reveals 'Unprecedented Results' From Rare Neurological Disease Trial

Ionis Pharmaceuticals, Inc. IONS released topline results on Monday from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive, and often fatal neurological condition.The neurological disorder destroys the myelin sheath protecting nerve fibers, caused by a mutation in the GFAP gene.Zilganersen 50 mg demonstrated statistically significant and clinically meaningful stabilization on the primary endpoint of gait speed as assessed by the 10-Meter Walk Te ...