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Updated: Feb 17, 2026

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
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试验面试,以探索糖原储存疾病型 Ia 患者经验 基因治疗后的经验

Diane M Turner-Bowker1, Jessica Butler2, Shayna Egan3

  • 1Ultragenyx Pharmaceutical, Inc., Novato, California, USA.

Journal of health economics and outcomes research
|February 16, 2026
PubMed
概括

基因疗法DTX401显著改善了Glycogen存储疾病类型Ia (GSDIa) 患者的症状,并减少了患者的日常负担. 大多数参与者报告说,他们的健康和生活质量有所改善,尽管面临小挑战,但他们的满意度很高.

关键词:
退出面试 退出面试基因治疗的基因疗法糖原储存疾病 糖原储存疾病糖原储存疾病类型 Ia Ia.与健康相关的生活质量.质量面试 质量面试 质量面试

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