林业发展局批准了Neurizon的NUZ-001号试验试验实验性ALS新疗法。 The FDA approved Neurizon’s NUZ-001 for a major ALS trial testing new treatments.

林业发展局已批准Neurizon治疗技术NUZ-001进入HEALY ALS平台试验,这是一个多中心适应性研究测试潜在ALS治疗方法。 The FDA has approved Neurizon Therapeutics’ NUZ-001 to enter the HEALEY ALS Platform Trial, a multicenter, adaptive study testing potential ALS treatments. 该药物以TDP-43集成和自发功能受损为目标,在早期数据中显示出有希望的安全性和大脑渗透。 The drug, which targets TDP-43 aggregation and impaired autophagy, showed promising safety and brain penetration in early data. 预计2026年初将开始现场激活和病人登记。 Site activations and patient enrollment are expected to begin in early 2026. 该试验由Mass General Brigham和NEALS牵头,目的是通过同时测试多种疗法来加快药物研制。 The trial, led by Mass General Brigham and NEALS, aims to speed up drug development by testing multiple therapies simultaneously. NUZ-001号仍在调查中,尚未核准出售。 NUZ-001 remains investigational and is not yet approved for sale.