Ionis Opening Phase 3 Trial of ION363, Antisense Therapy for FUS-ALS

Ionis Opening Phase 3 Trial of ION363, Antisense Therapy for FUS-ALS

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Ionis Opening Phase 3 Trial of ION363, Antisense Therapy for FUS-ALS

Ionis Pharmaceuticals is opening a Phase 3 safety and efficacy trial of ION363 (jacifusen) in amyotrophic lateral sclerosis (ALS) patients with confirmed mutations in the FUS gene, a known cause of juvenile-onset disease. The trial (NCT04768972) will enroll up to 64 people, ranging in age from 12 to 65, who are not on permanent ventilation. It is expected to begin recruitment this month; information on contacts and locations will be available here. “There is an urgent need…

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