一种被遗忘的药物为罕见疾病的儿童带来新希望
一种几十年前的药物曾用于治疗睡眠病，现在正在对一种罕见且致命的遗传性疾病——巴赫曼-布普综合征（BABS）展现出令人惊讶的希望。早期的患者治疗结果表明，这种药物DFMO可能通过针对基因突变来缓解严重症状。研究人员已经对几名患者进行了…
来源: ScienceDaily ｜ 阅读全文
发布时间: 2026-04-29 13:28 GMT+8（北京时间）
#DFMO #巴赫曼布普综合征 #遗传性疾病
Post #4833
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