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Child with ASMD — official photo, Lecheng archive
🇨🇳 China中国 Official story · Dec 2024官方故事 · 2024 年 12 月

A two-year hunt for one rare drug, and the relay that ended in Lecheng孩子确诊这种罕见病后,一场多方接力的“寻药”马拉松在乐城“开跑”

Patient患者
Child with ASMD酸性鞘磷脂酶缺乏症患儿
Condition病种
Acid sphingomyelinase deficiency (ASMD)酸性鞘磷脂酶缺乏症(ASMD)
Treatment治疗
Olipudase alfa, accessed through Lecheng经乐城用上奥立扑酶α
Outcome结果
Two years of searching for a drug that was not sold in China两年寻药,终于找到国内买不到的药

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。

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Individual case, not a clinical study and not a promise about your outcome. Treatment decisions belong to you and your doctor. Photos © the Lecheng Administration, used with credit; this page summarises the published facts in our own words — original article. 个案,非临床研究,不构成对你治疗结果的承诺。治疗决定由你和你的医生做出。照片版权归Lecheng Administration所有并已标注来源;本页以我们自己的文字概述已发布的事实——官方原文。