Ionis today has announced that they have commenced a pivotal clinical study of ION373 – an investigational antisense medicine designed to reduce the level of GFAP – in patients with Alexander disease.
Alexander disease patients experience progressive deterioration, leading to severe disability and loss of independence. Childhood onset is associated with more serious disease, with patients rarely surviving beyond adolescence. Alexander disease is caused by a genetic mutation that leads to overproduction and toxic accumulation of glial fibrillary acidic protein (GFAP) in the brain. ION373 is an investigational antisense medicine designed to reduce the level of GFAP.
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