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CAS No.: 1825352-65-5
Synonyms: RG7916;RO7034067;RO703406
Risdiplam is an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier that increases survival motor neuron (SMN) protein levels.
生物活性
动物研究 | ||||||||||||||||||||||
剂量 | Mice: 1 mg/kg - 10 mg/kg[1] (p.o.) Cynomolgus monkey: 0.1 mg/kg, 1.9 mg/kg[1] (i.v.); 0.5 mg/kg, 5.5 mg/kg[1] (p.o.) | |||||||||||||||||||||
给药途径 | p.o., i.v. | |||||||||||||||||||||
动物药代数据 |
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NCT号 | 适应症或疾病 | 临床期 | 招募状态 | 预计完成时间 | 地点 |
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NCT03040635 | Healthy Volunteers | Phase 1 | Completed | - | United States, California ... more >> Collaborative Neuroscience Network, Inc. Garden Grove, California, United States, 92845 Collapse << |
NCT02908685 | Muscular Atrophy, Spinal | Phase 2 Phase 3 | Active, not recruiting | July 12, 2020 | - |
NCT02633709 | Spinal Muscular Atrophy | Phase 1 | Completed | - | Netherlands ... more >> Pra International Group B.V Groningen, Netherlands, 9728 NZ Collapse << |
实验方案
技术信息
CAS号 | 1825352-65-5 | 储存条件 |
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分子式 | C22H23N7O | 运输 | 蓝冰 | |
分子量 | 401.46 | 别名 | RG7916;RO7034067;RO703406 |
NCT号 | 适应症或疾病 | 临床期 | 招募状态 | 预计完成时间 | 地点 |
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NCT03040635 | Healthy Volunteers | Phase 1 | Completed | - | United States, California ... more >> Collaborative Neuroscience Network, Inc. Garden Grove, California, United States, 92845 Collapse << |
NCT02908685 | Muscular Atrophy, Spinal | Phase 2 Phase 3 | Active, not recruiting | July 12, 2020 | - |
NCT02633709 | Spinal Muscular Atrophy | Phase 1 | Completed | - | Netherlands ... more >> Pra International Group B.V Groningen, Netherlands, 9728 NZ Collapse << |
NCT03036501 | Healthy | Phase 1 | Completed | - | Netherlands ... more >> Pra International Group B.V Groningen, Netherlands, 9728 NZ Collapse << |
NCT02913482 | Muscular Atrophy, Spinal | Phase 2 Phase 3 | Active, not recruiting | September 1, 2020 | - |
NCT03032172 | Spinal Muscular Atrophy | Phase 2 | Recruiting | December 1, 2020 | United States, New York ... more >> Columbia University Medical Center; The Neurological Institute of New York Recruiting New York, New York, United States, 10032 Italy Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile Recruiting Roma, Lazio, Italy, 00168 Switzerland Universitäts-Kinderspitalbeider Basel_Abteilung für Neuro- und Entwicklungspädiatrie Recruiting Basel, Switzerland, 4005 Collapse << |
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