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Pack Size | Price | Availability | Quantity |
---|---|---|---|
5 mg | $108 | Backorder | |
10 mg | $171 | Backorder | |
50 mg | $584 | Backorder | |
100 mg | $873 | Backorder | |
200 mg | Inquiry | Backorder | |
500 mg | Inquiry | Backorder |
Description | RG7800 hydrochloride is an orally active modulator of SMN2 splicing. |
Targets&IC50 | SMN2 splicing: 87 nM , SMN protein:, EC1.5x: 23 nM |
In vitro | RG7800 hydrochloride is an orally active modulator of SMN2 splicing(EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein).RG7800 hydrochloride is the first small molecule SMN2 splicing modifier to enter human clinical trials. |
In vivo | RG7800 dose-dependently elevates the SMN protein level in the brain and in peripheral tissue of Δ7 mice. RG7800 is a favorable agent metabolism and pharmacokinetic (DMPK) profile in the rat and in cynomolgus monkey with good oral bioavailability. |
Alias | RO6885247 hydrochloride, RG7800 hydrochloride |
Formula | C24H28N6O.xHCl |
Cas No. |
Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. |
Solubility Information | H2O: 113.5 mg/mL |
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