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

High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial

高剂量诺西那生治疗脊髓性肌萎缩症:一项3期随机试验

Finkel, Richard S; Crawford, Thomas O; Mercuri, Eugenio; Sumner, Charlotte J; Garcia Romero, Maria Del Mar; Day, John W; Montes, Jacqueline; Sun, Peng; Tichler, Ben; Paradis, Angela D; Boesch, Emily; Inra, Jennifer; Littauer, Ross; Sohn, Jihee; Monine, Michael; Gambino, Giulia; Foster, Richard; Farewell, Raechel; Fradette, Stephanie

Long-Term Tofersen in SOD1 Amyotrophic Lateral Sclerosis

托弗森治疗SOD1肌萎缩侧索硬化症的长期疗效

Miller, Timothy M; Cudkowicz, Merit E; Shaw, Pamela J; Genge, Angela; Sobue, Gen; Bucelli, Robert C; Chiò, Adriano; Van Damme, Philip; Ludolph, Albert C; Glass, Jonathan D; Andrews, Jinsy A; Babu, Suma; Benatar, Michael; McDermott, Christopher J; Salachas, François; Bruneteau, Gaëlle; Al-Chalabi, Ammar; Amorin, Matthew; Nestorov, Ivan; Graham, Danielle; Lin, Luan; Sun, Peng; McNeill, Manjit; Malek, Sohail; Inra, Jennifer; Garafalo, Steve; Fradette, Stephanie

Identification of tofersen PD-response biomarkers in VALOR clinical trial CSF via multiplexed quantitative proteomics.

通过多重定量蛋白质组学鉴定 VALOR 临床试验脑脊液中托福生帕金森反应生物标志物。

Guise Amanda J, Ferber Kyle L, Young Damon, Edwards Amanda L, Sabouri Somayeh, Fraser Kyle B, Milliman Eric, Plowey Edward D, Zoghbi Jad, Fradette Stephanie M, Graham Danielle L

Central Nervous System Biodistribution and Pharmacokinetics of Radiolabeled Tofersen in Rodents, Nonhuman Primates, and Humans

放射性标记的托福生在啮齿动物、非人灵长类动物和人类中枢神经系统的生物分布和药代动力学

Cook, Brendon E; McLaren, Donald G; Sullivan, Jenna M; El Fakhri, Georges; Yokell, Daniel L; Freeman, Mason W; Currier, Nicolas; Oestergaard, Michael E; Dobson, Howard; Hesterman, Jacob; Salem, Nicolas; Nestorov, Ivan; Monine, Michael; Martarello, Laurent; Evans, Karleyton C; Fradette, Stephanie; Ferguson, Toby A; Graham, Danielle; Passamonti, Luca

Operationalizing the environmental safe operating space into target distributions for mobility and batteries

将环境安全运行空间转化为移动性和电池的目标分布。

Roy, Sophia; Ali, Abdur-Rahman; Harvey, Jean-Philippe; Herrmann, Christoph; Fradette, Louis

Deferiprone in Alzheimer Disease: A Randomized Clinical Trial

去铁酮治疗阿尔茨海默病:一项随机临床试验

Ayton, Scott; Barton, David; Brew, Bruce; Brodtmann, Amy; Clarnette, Roger; Desmond, Patricia; Devos, David; Ellis, Kathryn A; Fazlollahi, Amir; Fradette, Caroline; Goh, Anita M Y; Kalinowski, Pawel; Kyndt, Christopher; Lai, Rosalyn; Lim, Yen Ying; Maruff, Paul; O'Brien, Terence J; Rowe, Christopher; Salvado, Olivier; Schofield, Peter W; Spino, Michael; Tricta, Fernando; Wagen, Aaron; Williams, Robert; Woodward, Michael; Bush, Ashley I

Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy

一项开放标签的IV期试验评估了在脊髓性肌萎缩症患儿中,接受onasemnogene abeparvovec治疗后使用nusinersen的疗效。

Proud, Crystal M; Finkel, Richard S; Parsons, Julie A; Masson, Riccardo; Brandsema, John F; Kuntz, Nancy L; Foster, Richard; Li, Wenjing; Littauer, Ross; Sohn, Jihee; Fradette, Stephanie; Youn, Bora; Paradis, Angela D

Landscapes of missense variant impact for human superoxide dismutase 1

人类超氧化物歧化酶1错义变异影响图谱

Axakova, Anna; Ding, Megan; Cote, Atina G; Subramaniam, Radha; Senguttuvan, Vignesh; Zhang, Haotian; Weile, Jochen; Douville, Samuel V; Gebbia, Marinella; Al-Chalabi, Ammar; Wahl, Alexander; Reuter, Jason; Hurt, Jessica; Mitchell, Adele A; Fradette, Stephanie; Andersen, Peter M; van Loggerenberg, Warren; Roth, Frederick P

Multi-Year Registry Study of Elapegademase Treatment in Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Requiring Enzyme Replacement Therapy

一项针对需要酶替代疗法的腺苷脱氨酶重症联合免疫缺陷病(ADA-SCID)患者的Elapegademase治疗多年注册研究

Dorsey, Morna J; Butte, Manish J; Lieberman, Jay A; Lehman, Heather; Fausnight, Tracy; Keller, Michael D; Fradette, Caroline; Hershfield, Michael S; Pozos, Tamara C; Rozova, Anna; Wall, Luke A; Bednarski, Jeffrey J; Tarrant, Teresa K; Chong, Hey J; Geng, Bob; Temin, Noemi Toiber; Laubach, Susan S; Lin, Leo; Mousallem, Talal; Walter, Jolan E

Correction of Significant Urethral Anomalies Using a Tissue-Engineered Human Urethral Substitute: Proof of Concept.

利用组织工程化人类尿道替代物矫正重大尿道畸形:概念验证

Caneparo Christophe, Elia Elissa, Chabaud Stéphane, Berthod François, Fradette Julie, Bolduc Stéphane