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SMN-C3 (MV8T2MCK57) is an orally active modulator of SMN2 splicing, and has the potential to treat spinal muscular atrophy (SMA).
Pack Size | Price | Availability | Quantity |
---|---|---|---|
1 mg | $179 | In Stock | |
5 mg | $455 | In Stock | |
10 mg | $672 | In Stock | |
25 mg | $1,060 | In Stock | |
50 mg | $1,430 | In Stock | |
100 mg | $1,920 | In Stock | |
500 mg | $3,850 | In Stock |
Description | SMN-C3 (MV8T2MCK57) is an orally active modulator of SMN2 splicing, and has the potential to treat spinal muscular atrophy (SMA). |
In vivo | At P16, vehicle-treated D7 mice were significantly smaller and dying compared to heterozygous control littermates. In contrast, D7 mice treated with high doses of SMN-C3 exhibited phenotypes similar to the heterozygous controls. SMN-C3 treatment in D7 mice induces dose-dependent body weight gain, with some achieving ~80% of heterozygous control weight. SMN-C3 also normalizes D7 motor behavior, allowing rapid self-righting and comparable locomotor activity to heterozygous controls. Importantly, vehicle-treated mice die within 3 weeks post-birth (median survival of 18 days), whereas SMN-C3 extends survival dose-dependently, with a median of 28 days at low dose (0.3 mg/kg/day) and ~90% survival beyond P65 in the higher doses (1 and 3 mg/kg/day) [1]. |
Alias | MV8T2MCK57 |
Molecular Weight | 416.52 |
Formula | C24H28N6O |
Cas No. | 1449597-34-5 |
Smiles | CCN1CCC(CC1)c1cc(C)c2nc(cc(=O)n2c1)-c1cc2c(C)nc(C)cn2n1 |
Relative Density. | 1.31 g/cm3 (Predicted) |
Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. | ||||||||||
Solubility Information | DMSO: 2.0 mg/mL (4.8 mM), Sonication and heating to 60℃ are recommended. | ||||||||||
Solution Preparation Table | |||||||||||
DMSO
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