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预防DMD基因疗法引发过度免疫反应:Imlifidase获得可喜早期试验数据
DMD是一种罕见且致命的遗传性疾病,由编码抗肌营养不良蛋白的DMD基因突变引起。这是一种不可逆的进行性疾病,会导致身体肌肉随着时间的推移而变得虚弱和受损,大多数患者在12岁之前就不得不依靠轮椅生活。在DMD患者中,约14%的患者体内已存在针对rh74 ...
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