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RG7800 is a small molecule SMN2 splicing modifier. RG7800 has the potential to be used in spinal muscular atrophy. RG7800 corrects selective splicing of the human SMN2 gene in the brain of a transgenic SMA model mouse, leading to an increase in SMN protein in the brain.
| Molecular Weight | 416.52 |
| Formula | C24H28N6O |
| CAS Number | 1449598-06-4 |
| Solubility (25°C) | DMSO：1.4 mg/mL |
| Storage | 4-8°C |
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