Brampton两岁幼儿Zoravar Singh患芳香族L-氨基酸脱羧酶缺乏症,家庭希望前往Boston接受Kebilidi基因疗法。OMNI报道,家属筹款目标约550万加元,已筹逾160万元;这属于报道当时的筹款进度,不是实时总额。

美国FDA于2024年11月批准Kebilidi用于该罕见遗传病。加拿大与美国批准、资助是不同程序:报道指该疗法尚未提交加拿大卫生部审批,安省因此未批准家庭的境外资助申请。

家属表示希望改善孩子发展能力,正寻求社会支持。美国批准不代表每位患者都会达到相同效果,也不能把治疗称为保证治愈;是否适合、风险及后续照护须由治疗团队评估。

来源:CityNews Toronto 原报道。大鱼新闻整理原创中文摘要;保留原刊发日期。

相关原始报告

The family of two-year-old Zoravar Singh is asking for help for treatment of his rare condition after being denied out-of-country coverage. HANDOUT/Singh family

The family of two-year-old Zoravar Singh is asking for help for treatment of his rare condition after being denied out-of-country coverage. HANDOUT/Singh family