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作用机制DMD exon 51 inhibitors [+2] |
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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- |
最高研发阶段药物发现 |
首次获批国家/地区- |
首次获批日期1800-01-20 |
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension (LTE) period (192 weeks).
100 项与 TfR1 x DMD exon 51 相关的临床结果
100 项与 TfR1 x DMD exon 51 相关的转化医学
0 项与 TfR1 x DMD exon 51 相关的专利(医药)