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A Pediatric Concussion Model in Mice: Closed Head Injury with Long-Term Disorders (CHILD)
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探讨长期使用安布罗克索在高氏病类型2中的长期使用:来自两个儿科病例的见解

Charlotte Aries1, Anja Köhn1, Karolin Täuber1

  • 1International Center for Lysosomal Disorders, Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.

Frontiers in neurology
|February 9, 2026
PubMed
概括

安布罗克索尔 (ABX) 显示出作为一种依赖于变异的治疗方法来治疗高氏病2型 (GD2),一种严重的神经疾病. 在两名患者的长期治疗中,酶活性和发育进展有所改善,为这种罕见的疾病提供了希望.

关键词:
戈舍氏病2型 2型的疾病.酶替代疗法是一种酶替代疗法.高剂量的安布罗克索尔.溶酶体储存障碍 溶酶体储存障碍神经病的高希氏病.

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