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

The Placebo Effect in Rare Disease Clinical Trials: Measurement, Impact, and Statistical Approaches for Patient-as-Own-Control Designs

罕见病临床试验中的安慰剂效应:以患者自身对照设计为例的测量、影响和统计方法

Summar, Marshall L; Woodcock, Janet

A novel, high throughput, and low-cost method for the detection of 40 amines relevant to inborn errors of metabolism, in under 60 min, using reverse phase high performance liquid chromatography

一种新型、高通量、低成本的检测方法,利用反相高效液相色谱法,可在60分钟内检测40种与先天性代谢缺陷相关的胺类物质。

Wilson, Kirkland A; Zhou, Yun; Cunningham, Gary; Chapman, Kimberly; Summar, Marshall; Regier, Debra

Personalized Ophthalmic Anesthesia: A Regression Analysis of Patient Characteristics, Surgical Profiles, and Anesthesia Protocols for Outcome Prediction

个性化眼科麻醉:基于患者特征、手术情况和麻醉方案的回归分析以预测手术结果

Shahzadi, Iram; Fatima, Summar; Ameen, Samreen; Atta, Asma; Atta, Maryam; Batool, Syeda W; Aslam, Rehan; Khan, Marriam

Survival and Prognostic Determinants after IMRT and 3D Radiotherapy in Metachronus Bone - Only Spinal Metastasis of Breast Cancer: A Prospective Phase II Trial

IMRT 和 3D 放射治疗对异时性骨转移(仅脊柱转移性乳腺癌)患者的生存率和预后决定因素:一项前瞻性 II 期试验

Youssif, Ayatallah; Abdellah, Asmaa; El-Morshidy, Summar; Hasaballah, Asmaa

mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria

mRNA疗法可纠正临床前精氨酸琥珀酸尿症中谷胱甘肽代谢缺陷并恢复尿素生成。

Gurung, Sonam; Timmermand, Oskar Vilhelmsson; Perocheau, Dany; Gil-Martinez, Ana Luisa; Minnion, Magdalena; Touramanidou, Loukia; Fang, Sherry; Messina, Martina; Khalil, Youssef; Spiewak, Justyna; Barber, Abigail R; Edwards, Richard S; Pinto, Patricia Lipari; Finn, Patrick F; Cavedon, Alex; Siddiqui, Summar; Rice, Lisa; Martini, Paolo G V; Ridout, Deborah; Heywood, Wendy; Hargreaves, Ian; Heales, Simon; Mills, Philippa B; Waddington, Simon N; Gissen, Paul; Eaton, Simon; Ryten, Mina; Feelisch, Martin; Frassetto, Andrea; Witney, Timothy H; Baruteau, Julien

The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria

运动障碍的发生率随年龄增长而增加,这与精氨酸琥珀酸尿症中轻微且有限的神经影像学异常形成对比。

Gurung, Sonam; Karamched, Saketh; Perocheau, Dany; Seunarine, Kiran K; Baldwin, Tom; Alrashidi, Haya; Touramanidou, Loukia; Duff, Claire; Elkhateeb, Nour; Stepien, Karolina M; Sharma, Reena; Morris, Andrew; Hartley, Thomas; Crowther, Laura; Grunewald, Stephanie; Cleary, Maureen; Mundy, Helen; Chakrapani, Anupam; Batzios, Spyros; Davison, James; Footitt, Emma; Tuschl, Karin; Lachmann, Robin; Murphy, Elaine; Santra, Saikat; Uudelepp, Mari-Liis; Yeo, Mildrid; Finn, Patrick F; Cavedon, Alex; Siddiqui, Summar; Rice, Lisa; Martini, Paolo G V; Frassetto, Andrea; Heales, Simon; Mills, Philippa B; Gissen, Paul; Clayden, Jonathan D; Clark, Christopher A; Eaton, Simon; Kalber, Tammy L; Baruteau, Julien

Ex vivo precision-cut liver slices model disease phenotype and monitor therapeutic response for liver monogenic diseases

离体精确切割的肝切片可模拟疾病表型并监测肝脏单基因疾病的治疗反应

Dany Perocheau, Sonam Gurung, Loukia Touramanidou, Claire Duff, Garima Sharma, Neil Sebire, Patrick F Finn, Alex Cavedon, Summar Siddiqui, Lisa Rice, Paolo G V Martini, Andrea Frassetto, Julien Baruteau

Pharmacological Progress of Mitophagy Regulation

线粒体自噬调控的药理学进展

Sehgal, Sheikh Arslan; Wu, Hao; Sajid, Muhammad; Sohail, Summar; Ahsan, Muhammad; Parveen, Gulnaz; Riaz, Mehreen; Khan, Muhammad Saleem; Iqbal, Muhammad Nasir; Malik, Abbeha

Synthetic mRNA rescues very long-chain acyl-CoA dehydrogenase deficiency in patient fibroblasts and a murine model

合成mRNA可挽救患者成纤维细胞和鼠模型中的极长链酰基辅酶A脱氢酶缺乏症。

Zhao, Xue-Jun; Mohsen, Ai-Walid; Mihalik, Stephanie; Solo, Keaton; Aliu, Ermal; Shi, Huifang; Basu, Shakuntala; Kochersperger, Catherine; Van't Land, Clinton; Karunanidhi, Anuradha; Coughlan, Kimberly A; Siddiqui, Summar; Rice, Lisa M; Hillier, Shawn; Guadagnin, Eleonora; Giangrande, Paloma H; Martini, Paolo G V; Vockley, Jerry

Messenger RNA rescues medium-chain acyl-CoA dehydrogenase deficiency in fibroblasts from patients and a murine model

信使RNA可挽救患者和鼠模型成纤维细胞中的中链酰基辅酶A脱氢酶缺乏症

Zhao, Xue-Jun; Mohsen, Ai-Walid; Mihalik, Stephanie; Solo, Keaton; Basu, Shakuntala; Aliu, Ermal; Shi, Huifang; Kochersberger, Catherine; Karunanidhi, Anuradha; Van't Land, Clinton; Coughlan, Kimberly A; Siddiqui, Summar; Rice, Lisa M; Hillier, Shawn; Guadagnin, Eleonora; DeAntonis, Christine; Giangrande, Paloma H; Martini, Paolo G V; Vockley, Jerry