AT-007 是口服有效的，中枢神经系统渗透性醛糖还原酶的高效抑制剂， IC50为 100 pM。在 GALT 缺失大鼠中，它降低了有毒的半乳糖醇水平并预防了疾病并发症。

AT-007 is an orally active CNS penetrant Aldose Reductase inhibitor for the treatment of Galactosemia (IC50: 100 pM). It reduces toxic galactitol levels and prevents disease complications in GALT deficiency rats.

AT-007 significantly reduces galactitol levels in GALT null rats in all target tissues ( liver, brain, and plasma), and does not increase galactose or Gal-1P levels [2].

2170729-29-8

C17H10F3N3O3S2

425.41

DMSO:35.71 mg/mL (83.94 mM),Need ultrasonic
Powder: -20°C for 3 years
In solvent: -80°C for 2 years