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Several regulatory\u2010led initiatives have progressed rapidly during the past year, including patient\u2010focused drug development, model\u2010informed drug development, real\u2010world evidence, and complex innovative trial designs. Collectively, these initiatives have accelerated the rate of approvals. Despite demands to focus on urgent needs imposed by the COVID\u201019 pandemic, the number of new drug approvals over the past year, particularly for rare diseases, has outpaced expectations. Advancing therapeutics for nervous system disorders requires adaptive strategies that align with rapid developments in the field. Three relentlessly progressive diseases, amyotrophic lateral sclerosis, Duchenne muscular dystrophy, and Parkinson\u2019s disease are in urgent need of new treatments. 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