包装 | 价格(元) |
5mg | 电议 |
10mg | 电议 |
50mg | 电议 |
100mg | 电议 |
200mg | 电议 |
500mg | 电议 |
生物活性 | RG7800 hydrochloride is an orally activeSMN2splicing modulator, withEC1.5xs of 23 nM and 87 nM forSMN2splicing andSMN protein; RG7800 hydrochloride has the potential to treat spinal muscular atrophy. |
IC50& Target | EC1.5x: 23 nM (SMN2splicing), 87 nM (SMN protein)[1] |
体外研究 (In Vitro) | RG7800 hydrochloride is an orally activeSMN2splicing modulator, with EC1.5xs of 23 nM and 87 nM forSMN2splicing and SMN protein; and is the first small moleculeSMN2splicing modifier to enter human clinical trials[1]. |
体内研究 (In Vivo) | RG7800 is a favorable agent metabolism and pharmacokinetic (DMPK) profile in the rat and in cynomolgus monkey with good oral bioavailability. RG7800 (1, 3, 10 mg/kg, p.o.) dose-dependently elevates the SMN protein level in the brain and in peripheral tissue of Δ7 mice[1]. |
Clinical Trial | |
性状 | Solid |
Formula | C24H28N6O.xHCl |
运输条件 | Room temperature in continental US; may vary elsewhere. |
储存方式 | 4°C, sealed storage, away from moisture *In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture) |
溶解性数据 | In Vitro: H2O : ≥ 113.5 mg/mL *"≥" means soluble, but saturation unknown. |