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增加酶替代疗法的间隔,以治疗稳定的1型高氏病:一个非劣势性序列模拟试验

Maxime Beydon1,2, Jérôme Stirnemann3, Karima Yousfi1

  • 1Service de Médecine Interne, Centre de référence des maladies lysosomales, Filière maladies héréditaires du métabolisme, Hôpital Beaujon, AP-HP.Nord, Université Paris Cité, Paris, France.

Journal of internal medicine
|February 28, 2026
PubMed
概括

延长酶替代疗法 (ERT) 对于高氏病 (GD) 从每两周一次延长到每3-4周一次,对于稳定的患者来说是安全有效的. 这种变化保持了治疗的有效性,同时有可能改善生活质量和降低成本.

关键词:
戈舍氏病是戈舍氏病的一种疾病.酶替代疗法是一种酶替代疗法.溶酶体疾病是一种溶酶体疾病.

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