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Pack Size | Price | Availability | Quantity |
---|---|---|---|
2 mg | $42 | 5 days |
Description | RG7800 hydrochloride is an orally active modulator of SMN2 splicing, exhibiting EC50 values of 23 nM for SMN2 splicing and 87 nM for SMN protein production. This compound holds promise as a therapeutic agent for spinal muscular atrophy. |
In vitro | RG7800 hydrochloride is an orally active SMN2 splicing modulator that exhibits half-maximal effective concentrations (EC50s) of 23 nM for SMN2 splicing and 87 nM for SMN protein production. It holds the distinction of being the inaugural small molecule SMN2 splicing modifier to progress 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 |
Cas No. |
Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year |
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