For two years the parents of a boy called Gungun travelled the country looking for a way to obtain olipudase alfa, an enzyme replacement therapy for acid sphingomyelinase deficiency — a disease so rare that fewer than 120 patients have been diagnosed in the United States. Because ASMD needs lifelong treatment, flying abroad every few weeks was not a plan. The manufacturer had no near-term plan to enter China, so Lecheng’s special-access channel became the family’s route.
两年来,滚滚的父母跑遍全国,只为找到一款名为奥立扑酶α(Olipudase alfa)的药——它用于治疗酸性鞘磷脂酶缺乏症(ASMD),一种在美国确诊患者不足 120 例的极罕见病。因为需要终身用药,每隔几周飞国外并不现实;药企短期内也没有进入中国的计划。最终,乐城的特许通道成了这个家庭的出路。
This page is a short English/Chinese summary of the facts in a patient story published by the Lecheng Administration. Photos are from that publication, credited; a link to the original Chinese article sits at the foot of this page. 本页是对Lecheng Administration发布的一篇患者故事中事实的简短中英摘要。照片取自该发布并标注来源;官方中文原文链接见本页页脚。





