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  • Title: [Advances in the specific treatments of Gaucher disease].
    Author: Meng Y.
    Journal: Zhonghua Nei Ke Za Zhi; 2024 Jul 01; 63(7):702-711. PubMed ID: 38951097.
    Abstract:
    戈谢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。戈谢病的特异性治疗包括酶替代治疗、底物减少疗法、造血干细胞移植、分子伴侣疗法和基因治疗。目前酶替代治疗和底物减少疗法的药物已获批上市，其他新型治疗方案仍在临床研究中。本文综述并探讨了不同分型戈谢病特异性治疗的最新进展及不同分型患者特异性治疗的方案选择，以期为临床医生治疗该病提供参考。.
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