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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 | USA Warehouse | Global Warehouse | Quantity |
|---|---|---|---|---|
| 1 mg | $179 | In Stock | In Stock | |
| 5 mg | $455 | In Stock | In Stock | |
| 10 mg | $672 | In Stock | In Stock | |
| 25 mg | $1,060 | In Stock | In Stock | |
| 50 mg | $1,430 | In Stock | In Stock | |
| 100 mg | $1,920 | In Stock | 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]. |
| Synonyms | 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/Shipping at ambient temperature. | ||||||||||
| Solubility Information | DMSO: 2 mg/mL (4.8 mM), Sonication and heating to 60℃ are recommended. | ||||||||||
Solution Preparation Table | |||||||||||
DMSO
| |||||||||||
Dissolve 2 mg of the compound in 100 μL DMSO
to obtain a stock solution at a concentration of 20 mg/mL . If the required concentration exceeds the compound's known solubility, please contact us for technical support before proceeding.
1) Add 100 μL of the DMSO
stock solution to 400 μL PEG300
and mix thoroughly until the solution becomes clear.
2) Add 50 μL Tween 80 and mix well until fully clarified.
3) Add 450 μL Saline,PBS or ddH2O
and mix thoroughly until a homogeneous solution is obtained.
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