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RG7800 hydrochloride is an orally active SMN2 splicing modulator with EC50 values of 23 nM for splicing and 87 nM for SMN protein production, holding promise as a therapeutic agent for [spinal muscular atrophy].
Pack Size | Price | Availability | Quantity |
---|---|---|---|
2 mg | $42 | 5 days |
Description | RG7800 hydrochloride is an orally active SMN2 splicing modulator with EC50 values of 23 nM for splicing and 87 nM for SMN protein production, holding promise as a therapeutic agent for [spinal muscular atrophy]. |
In vitro | RG7800 hydrochloride is an orally active SMN2 splicing modulator with EC50s of 23 nM for SMN2 splicing and 87 nM for SMN protein production. It is the first small molecule SMN2 splicing modifier to advance into human clinical trials [1]. |
In vivo | RG7800 exhibits a favorable metabolism and pharmacokinetic (DMPK) profile in both rats and cynomolgus monkeys, paired with good oral bioavailability. Administered at doses of 1, 3, and 10 mg/kg orally, RG7800 dose-dependently increases SMN protein levels in both the central nervous system and peripheral tissues of Δ7 mice [1]. |
Alias | RO6885247 tetrahydrochloride |
Molecular Weight | 562.36 |
Formula | C24H32Cl4N6O |
Storage | Shipping with blue ice. |
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