Rare Neurologic Disease Treatment Milestones

FDA approved Ionis’s zilganersen for Alexander disease in adults and children after significant gait improvement at 61 weeks in the 50-mg group, creating an important option for a severe rare disorder. Broader functional benefit, safety, durability, and study-design details require follow-up. The first FDA-cleared MLIV gene-therapy trial is an additional early milestone, but preclinical support does not establish clinical efficacy.