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非在研适应症- |
最高研发阶段临床1期 |
首次获批国家/地区- |
首次获批日期- |
A Phase 1, Multicenter, Open-label Study to Investigate the Safety and Biodistribution of a Single Intrathecal Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (The ASCEND Study)
The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD.
100 项与 Insmed Gene Therapy LLC 相关的临床结果
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