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

Neurofilament Light Chain as a Biomarker of Disease Progression in Lafora Disease

神经丝轻链作为拉福拉病疾病进展的生物标志物

Muccioli, Lorenzo; Ganceviciute, Bazile; Becker, Felicitas; Minardi, Raffaella; Tappatà, Maria; Bachhuber, Franziska; Alkhatib, Mahmoud; Cirak, Sebahattin; Weishaupt, Jochen; Verma, Mayank; Tumani, Hayrettin; Wagner, Jan; Messahel, Souad; Nitschke, Felix; Minassian, Berge A; Bisulli, Francesca; Brenner, David

Fixed-duration ibrutinib-venetoclax for first-line treatment of patients with chronic lymphocytic leukemia: the REALITY-WW prospective real-world study cohort

一线治疗慢性淋巴细胞白血病患者的固定疗程伊布替尼-维奈克拉方案:REALITY-WW 前瞻性真实世界研究队列

Munir, Talha; Leão, Danielle; Messahel, Boo; Srikanthan, Sowmya; Xu, Ping; Fouad, Mohamed; Kavanagh, Claire; Tapprich, Christoph; Miles, Oliver; Scheliga, Adriana; Ghia, Paolo

The 9th annual Lafora science symposium: a rare epilepsy community makes progress towards clinical readiness

第九届拉福拉科学研讨会:罕见癫痫群体在临床准备方面取得进展

Williams, Meredith I; Donohue, Katherine J; Sanz, Pascual; Messahel, Souad; Serratosa, Jose M; Duran, Jordi; Michelucci, Roberto; Muccioli, Lorenzo; Delgado-Escueta, Antonio; Nguyen, Viet-Huong V; Minassian, Berge A; Gentry, Matthew S

AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient

AAV基因疗法治疗50型遗传性痉挛性截瘫:一项单例患者的I期临床试验

Dowling, James J; Pirovolakis, Terry; Devakandan, Keshini; Stosic, Ana; Pidsadny, Mia; Nigro, Elisa; Sahin, Mustafa; Ebrahimi-Fakhari, Darius; Messahel, Souad; Varadarajan, Ganapathy; Greenberg, Benjamin M; Chen, Xin; Minassian, Berge A; Cohn, Ronald; Bonnemann, Carsten G; Gray, Steven J

(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

Seizure control via pH manipulation: a phase II double-blind randomised controlled trial of inhaled carbogen as adjunctive treatment of paediatric convulsive status epilepticus (Carbogen for Status Epilepticus in Children Trial (CRESCENT))

通过 pH 值调节控制癫痫发作:吸入卡波根作为儿童惊厥性癫痫持续状态辅助治疗的 II 期双盲随机对照试验(儿童惊厥持续状态卡波根试验 (CRESCENT))

Forsyth, Rob; Allen, Maria; Bedson, Emma; Downes, Alice; Gough, Chris; Hartshorn, Stuart; Lawton, Kelly; Lyttle, Mark D; Messahel, Shrouk; Mullen, Niall; Raper, Joseph; Rosala-Harris, Anna; Taggart, Leigh; Urron, Jason; Walton, Emily; Gamble, Carrol

Molecular Evidence of Spotted Fever Group Rickettsiae in Ticks and Fleas Collected from Some Wild and Domestic Animals in Different Biotopes in Algeria

阿尔及利亚不同生境中一些野生和家养动物身上采集的蜱虫和跳蚤中斑疹热群立克次体的分子证据

Beneldjouzi, Assia; Lafri, Ismail; Djerbouh, Amel; Nekhili, Safia; Hamrioui, Lydia; Messahel, Nacer Eddine; Harrat, Zoubir; Eddaikra, Naouel; Bitam, Idir

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