| Description | PTC258 is a specific, orally active modulator of the splicing of the Elongator complex protein 1 gene (ELP1), enhancing its expression both in vitro and in vivo. Additionally, PTC258 has demonstrated good tolerability in a mouse model [1]. |
| In vivo | PTC258（3-24 mg/kg；口服；每天一次，连续3个月）具有良好的耐受性。它能够纠正ELP1转录本的剪接，并在小鼠模型中显著提高所有测试组织内，包括大脑中的功能性蛋白质含量[1]。 |
| molecular weight | 351.92 |
| Molecular formula | C16H18ClN3S2 |
| CAS | 2476724-74-8 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year |