Venglustat在试验第3阶段第3类Gaucher病的试验中满足了所有初级和关键二级端点,显示出了较高的神经效益和口服方便。 Venglustat met all primary and key secondary endpoints in a phase 3 trial for type 3 Gaucher disease, showing superior neurological benefits and oral convenience.

Sanofi报告说,Venglustat在其第三阶段LEAP2MONO对12岁和12岁以上的病人的3类Gaucher疾病进行LEAP2MONO试验时,满足了所有初级和4个关键二级端点中的3个端点,表明静脉酶替代疗法和较高的神经结果具有类似的非遗传效益。 Sanofi reported that venglustat met all primary and three of four key secondary endpoints in its phase 3 LEAP2MONO trial for type 3 Gaucher disease in patients aged 12 and older, showing comparable non-neurological benefits to intravenous enzyme replacement therapy and superior neurological outcomes. 口服毒品跨越血脑屏障,完全得到容忍,没有新的安全关切。 The oral drug, which crosses the blood-brain barrier, was well tolerated with no new safety concerns. Sanofi计划提交全球监管报告,并强调每日避孕药有可能改变这种罕见神经紊乱的治疗方式。 Sanofi plans global regulatory submissions and highlighted the potential of a daily pill to transform treatment for this rare neurological disorder.