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Ionis completes enrollment in pivotal trial evaluating zilganersen in people living with Alexander disease
IONSIonis Pharmaceuticals(IONS) Prnewswire·2024-07-18 11:00

Zilganersen is the first investigational medicine in clinical development for people with Alexander disease, a rare, life-threatening neurological condition AxD is estimated to occur in an estimated one in one million people in the U.S. and can present throughout life.1-3 The disease is a result of genetic variants in the glial fibrillary acidic protein (GFAP) gene that disrupt the structure and function of astrocytes in the brain. AxD is generally characterized by cognitive dysfunction and progressive neur ...