Abstract
庞贝病是一种多系统受累的罕见遗传性溶酶体贮积症，尽早治疗有助于延缓疾病进展，提高患者生活质量，延长患者预期寿命。庞贝病的特异性治疗包括酶替代治疗、基因治疗、分子伴侣疗法以及寡核苷酸药物等。目前只有酶替代治疗的药物已获批上市，其他新型治疗方案仍在临床研究中。本文介绍了庞贝病特异性治疗的最新进展，探讨了当前庞贝病患者特异性治疗方案的选择，以期为临床医生治疗该病提供参考。.
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