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非在研适应症- |
最高研发阶段临床1/2期 |
首次获批国家/地区- |
首次获批日期1800-01-20 |
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非在研适应症- |
最高研发阶段临床申请 |
首次获批国家/地区- |
首次获批日期1800-01-20 |
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非在研适应症- |
最高研发阶段临床前 |
首次获批国家/地区- |
首次获批日期1800-01-20 |
A Phase 1/2, First in Human, Multiple-dose, 2-part Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-050/NCNP-03 in Boys With Duchenne Muscular Dystrophy (DMD)
This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD mutation amenable to exon 50 skipping.
100 项与 DMD exon 50 相关的临床结果
100 项与 DMD exon 50 相关的转化医学
0 项与 DMD exon 50 相关的专利(医药)