日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

First-in-human high dose AAV9 intrathecal gene therapy for paediatric CLN7 disease: a phase 1, open-label, single ascending dose, non-randomised clinical trial

首次人体高剂量AAV9鞘内基因疗法治疗儿童CLN7疾病:一项1期、开放标签、单次递增剂量、非随机临床试验

Greenberg, Benjamin M; Minassian, Berge; Messahel, Souad; Edgar, Veronica Bordes; Lowden, Andrea; Dahshi, Hamza; Nettesheim, Emily R; Nguyen, Hoang H; Hughes, Samuel; Muthukumar, Alagar R; Srinivasan, Kalayarasan; Iannaccone, Susan; Varadarajan, Ganapathy; Gray, Steven J; Kayani, Saima N

Delta Power in SLC6A1-Related Neurodevelopmental Disorder: Operationalizing Quantitative EEG Metrics for Biomarker Development

SLC6A1相关神经发育障碍中的δ波功率:将定量脑电图指标应用于生物标志物开发

Dahshi, Hamza; Varnet, Marie; Goodspeed, Kimberly; Tiller, Jacob; Armstrong, Dallas; Sirsi, Deepa

Serum predictors of native liver survival post-Kasai: Systematic review and meta-analysis

Kasai手术后自体肝脏存活的血清预测因子:系统评价和荟萃分析

Anouti, Ahmad; Dahshi, Hamza; Cotter, Thomas G; Rabbani, Tebyan A; Corbitt, Natasha; Hassan, Sara

Racial Disparities in Pediatric Patients With Cholestatic Liver Disease Undergoing Liver Transplant: A UNOS Database Study

儿童胆汁淤积性肝病患者接受肝移植的种族差异:一项基于美国器官共享网络(UNOS)数据库的研究

Anouti, Ahmad; Dahshi, Hamza; Rady, Elias D; Cotter, Thomas G; Aqul, Amal; Bartlett, Johanna Ascher; Hassan, Sara; Pandurangi, Sindhu

Pediatric liver transplant outcomes: A comparative analysis of steatotic donor grafts

儿童肝移植结果:脂肪肝供体移植物的比较分析

Anouti, Ahmad; Dahshi, Hamza; Patel, Madhukar S; Cotter, Thomas G; Heimbach, Julie K; Hassan, Sara

Assessment of Hemophagocytic Lympho-Histiocytosis (HLH) in the Setting of Adult Acute Liver Failure.

成人急性肝衰竭背景下噬血细胞性淋巴组织细胞增生症(HLH)的评估

Anouti Ahmad, Dahshi Hamza, Rule Jody, Wysocki Christian, Lee William M, Tujios Shannan R

(1)H and (31)P magnetic resonance spectroscopy reveals potential pathogenic and biomarker metabolite alterations in Lafora disease

(1)H 和 (31)P 磁共振波谱揭示了拉福拉病中潜在的致病和生物标志物代谢物改变。

Chan, Kimberly L; Panatpur, Aparna; Messahel, Souad; Dahshi, Hamza; Johnson, Talon; Henning, Anke; Ren, Jimin; Minassian, Berge A

Neuronal ceroid lipofuscinoses type 7 (CLN7): a case series reporting cross sectional and retrospective clinical data to evaluate validity of standardized tools to assess disease progression, quality of life, and adaptive skills

神经元蜡样脂褐质沉积症7型(CLN7):一项病例系列研究,报告横断面和回顾性临床数据,以评估用于评估疾病进展、生活质量和适应能力的标准化工具的有效性

Kayani, Saima; BordesEdgar, Veronica; Lowden, Andrea; Nettesheim, Emily R; Dahshi, Hamza; Messahel, Souad; Minassian, Berge A; Greenberg, Benjamin M

Neuronal Ceroid Lipofuscinoses Type 7 (CLN7)- A Case Series Reporting Cross Sectional and Retrospective Clinical Data to Evaluate Validity of Standardized Tools to Assess Disease Progression, Quality of Life, and Adaptive Skills

神经元蜡样脂褐质沉积症 7 型 (CLN7) - 病例系列报告:横断面和回顾性临床数据,以评估用于评估疾病进展、生活质量和适应技能的标准化工具的有效性

Kayani, Saima; Edgar, Veronica Bordes; Lowden, Andrea; Nettesheim, Emily R; Dahshi, Hamza; Messahel, Souad; Minassian, Berge A; Greenberg, Benjamin M