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相关临床试验
50
22 进行中
药物批准
0
批准总数
监管机构
监管机构数
成立时间
N/A
进行中（未招募）
2
4.0%
已完成
17
34.0%
尚未招募
20
40.0%
招募中
9
18.0%
终止
1
2.0%
撤回
暂无批准数据
- Skyhawk Therapeutics has dosed the first patient in its Phase 2/3 FALCON-HD trial evaluating SKY-0515, an oral RNA splicing modulator for Huntington's disease treatment. - SKY-0515 demonstrated promising Phase 1 results with up to 72% reduction in huntingtin mRNA in healthy volunteers and was generally well tolerated across all doses. - The investigational drug uniquely targets both huntingtin and PMS1 proteins, two key drivers of Huntington's disease pathology, potentially offering enhanced therapeutic impact. - The randomized, placebo-controlled trial will enroll 120 participants across 10 sites in Australia and New Zealand, representing a significant milestone for disease-modifying HD therapies.
