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T

Terry G. J. Derks

University of Groningen
H指数:33
被引数:3,641

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Phase 3 Randomized Trial Results of DTX401 AAV Gene Therapy for the Treatment of GSDIa

DTX401 AAV基因疗法治疗GSDIa的III期随机试验结果

2026-09-060
OAAI
Proteomics profiling of serum and liver in GSD ia and Ib patients: insights into complication mechanisms and circulation biomarkers

血清和肝脏的蛋白质组学分析在GSD ia和Ib患者中的应用:对并发症机制和循环生物标志物的见解

2026-02-200
OAAI
Pregnancies in Women With Long-Chain Fatty Acid Oxidation Disorders: Results of a European and North American Survey

患有长链脂肪酸氧化障碍的妇女的妊娠: 欧洲和北美的调查结果

2026-01-190
OAAI
Person-centered outcomes for liver glycogen storage diseases: development of an international consensus-based standard outcome set

以患者为中心的肝脏糖原贮积症结局:基于国际共识标准结局集的开发

2025-09-230
OAAI
Newborn Screening by DNA-First: Systematic Evaluation of the Eligibility of Inherited Metabolic Disorders Based on Treatability

DNA优先的新生儿筛查:基于可治疗性的遗传代谢病筛查资格的系统评价

2025-08-210
PRE
Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa)

DTX401的安全性和有效性评估,该疗法为AAV8介导的肝脏靶向基因治疗,用于治疗成人1型a型糖原贮积症(GSDIa)。

2025-03-101
OAAI
Newborn Screening by DNA-First: Systematic Evaluation of the Eligibility of Inherited Metabolic Disorders Based on Treatability
2024-12-280
OAAI
Precision-cut liver slices as an ex vivo model to assess impaired hepatic glucose production

精确切割肝切片作为体外模型评估受损的肝葡萄糖产生

2024-11-090
OAAI
Empagliflozin for treating neutropenia and neutrophil dysfunction in 21 infants with glycogen storage disease 1b

依帕列净治疗21例糖原贮积病1b婴儿的中性粒细胞减少和中性粒细胞功能障碍

2024-06-011
OAAI