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

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

Antecedent enhancer activity predicts future susceptibility to seizures in mice

前驱增强子活性可预测小鼠未来对癫痫的易感性

Boros, Benjamin D; Gachechiladze, Mariam A; Guo, Juanru; Galloway, Dylan A; Mueller, Shayna M; Shabsovich, Mark; Yen, Allen; Chen, Xuhua; Cammack, Alexander J; Shen, Tao; Mitra, Robi D; Dougherty, Joseph D; Miller, Timothy M

Neurofilament Proteoforms in Amyotrophic Lateral Sclerosis Are Different in Cerebrospinal Fluid and Blood

肌萎缩侧索硬化症患者的神经丝蛋白异构体在脑脊液和血液中存在差异

Coulton, John B; He, Yingxin; Budelier, Melissa M; Barthélemy, Nicolas; Ireland, Margaret D; Hu, Miwei; Graham, Danielle; Ferguson, Toby; Berry, James D; Bateman, Randall J; Miller, Timothy M; Ly, Cindy V

A quantitative cell-based reporter links TDP-43 aggregation and dysfunction to define pathogenic mechanisms

一种基于细胞的定量报告系统将TDP-43聚集和功能障碍联系起来,从而阐明其致病机制。

Mamede, Lohany Dias; Hu, Miwei; Vaquer-Alicea, Jaime; Titus, Amanda R; Passos, Patricia M; Lantelme, Erica; French, Rachel L; Kirschner, Paige A; Diamond, Marc I; Miller, Timothy M; Ayala, Yuna M

Pridopidine in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial

普利多吡啶治疗肌萎缩侧索硬化症:HEALEY ALS平台试验

Shefner, Jeremy M; Oskarsson, Björn; Macklin, Eric A; Chibnik, Lori B; Quintana, Melanie; Saville, Benjamin R; Detry, Michelle A; Vestrucci, Matteo; Marion, Joe; McGlothlin, Anna; Heiman-Patterson, Terry; Chase, Marianne; Pothier, Lindsay; Harkey, Brittney A; Yu, Hong; Sherman, Alexander V; Hall, Meghan; Kittle, Gale; Berry, James D; Babu, Suma; Andrews, Jinsy; D'Agostino, Derek; Tustison, Eric; Scirocco, Erica; Giacomelli, Elisa; Alameda, Gustavo; Locatelli, Eduardo; Ho, Doreen; Quick, Adam; Ajroud-Driss, Senda; Katz, Jonathan; Heitzman, Daragh; Appel, Stanley H; Shroff, Sheetal; Felice, Kevin; Maragakis, Nicholas J; Simmons, Zachary; Miller, Timothy M; Olney, Nicholas; Weiss, Michael D; Goutman, Stephen A; Fernandes, Joseph Americo; Jawdat, Omar; Owegi, Margaret Ayo; Foster, Laura A; Vu, Tuan; Ilieva, Hristelina; Newman, Daniel S; Arcila-Londono, Ximena; Jackson, Carlayne E; Ladha, Shafeeq; Caress, James B; Swenson, Andrea; Peltier, Amanda; Lewis, Richard A; Fee, Dominic; Elliott, Matthew; Bedlack, Richard; Kasarskis, Edward J; Elman, Lauren; Rosenfeld, Jeffrey; Walk, David; McIlduff, Courtney; Twydell, Paul; Young, Eufrosina; Johnson, Kristin; Rezania, Kourosh; Goyal, Namita A; Cohen, Jeffrey A; Benatar, Michael; Jones, Vovanti; Shah, Jaimin; Beydoun, Said R; Wymer, James P; Zilliox, Lindsay; Nayar, Shakti; Pattee, Gary L; Martinez-Thompson, Jennifer; Leitner, Melanie L; Chen, Kelly; Goldberg, Y Paul; Cohen, Yael; Geva, Michal; Hayden, Michael R; Paganoni, Sabrina; Cudkowicz, Merit E

Reldesemtiv in Amyotrophic Lateral Sclerosis: Results From the COURAGE-ALS Randomized Clinical Trial

Reldesemtiv治疗肌萎缩侧索硬化症:COURAGE-ALS随机临床试验结果

Shefner, Jeremy M; Cudkowicz, Merit E; Genge, Angela; Hardiman, Orla; Al-Chalabi, Ammar; Andrews, Jinsy A; Chio, Adriano; Corcia, Philippe; Couratier, Philippe; de Carvalho, Mamede; Heiman-Patterson, Terry; Henderson, Robert D; Ingre, Caroline; Johnston, Wendy; Ludolph, Albert; Maragakis, Nicholas J; Miller, Timothy M; Mora, Jesus S; Petri, Susanne; Simmons, Zachary; van den Berg, Leonard H; Zinman, Lorne; Kupfer, Stuart; Malik, Fady I; Meng, Lisa; Simkins, Tyrell J; Wei, Jenny; Wolff, Andrew A; Rudnicki, Stacy A

Network analysis of the cerebrospinal fluid proteome reveals shared and unique differences between sporadic and familial forms of amyotrophic lateral sclerosis

脑脊液蛋白质组网络分析揭示了散发型和家族型肌萎缩侧索硬化症之间的共同和独特差异

Trautwig, Adam N; Fox, Edward J; Dammer, Eric B; Shantaraman, Anantharaman; Ping, Lingyan; Duong, Duc M; Watson, Caroline M; Wu, Fang; Asress, Seneshaw; Guo, Qi; Levey, Allan I; Lah, James J; Verde, Federico; Doretti, Alberto; Ratti, Antonia; Ticozzi, Nicola; Ly, Cindy V; Miller, Timothy M; Garret, Mark A; Berry, James D; Thomas, Eleanor V; Fournier, Christina N; McEachin, Zachary T; Seyfried, Nicholas T; Glass, Jonathan D

Sporadic ALS induced pluripotent stem cell derived neurons reveal hallmarks of TDP-43 loss of function.

散发性 ALS 诱导多能干细胞衍生的神经元显示出 TDP-43 功能丧失的标志

Rothstein Jeffrey D, Keeley Olivia, Warlick Caroline, Miller Timothy M, Ly Cindy V, Glass Jonathan D, Coyne Alyssa N

Elucidating brain transport pathways and cell type-dependent gene silencing of a durable lipid-siRNA conjugate administered into cerebrospinal fluid.

阐明脑脊液中注入的持久性脂质-siRNA缀合物的脑运输途径和细胞类型依赖性基因沉默

Sorets Alexander G, Schwensen Katrina R, Francini Nora, Kjar Andrew, Abdulrahman Adam M, Shostak Alena, Katdare Ketaki A, Schoch Kathleen M, Cowell Rebecca P, Park Joshua C, Ligocki Alexander P, Ford William T, Ventura-Antunes Lissa, Hoogenboezem Ella N, Prusky Alex, Castleberry Mark, Michell Danielle L, Fritsch Emma, Lyons Sarah M, Miller Timothy M, Vickers Kasey C, Schrag Matthew S, Duvall Craig L, Lippmann Ethan S

Efficacy and Safety of Zilucoplan in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial

Zilucoplan治疗肌萎缩侧索硬化症的疗效和安全性:一项随机临床试验

Paganoni, Sabrina; Fournier, Christina N; Macklin, Eric A; Chibnik, Lori B; Quintana, Melanie; Saville, Benjamin R; Detry, Michelle A; Vestrucci, Matteo; Marion, Joe; McGlothlin, Anna; Ajroud-Driss, Senda; Chase, Marianne; Pothier, Lindsay; Harkey, Brittney A; Yu, Hong; Sherman, Alexander V; Shefner, Jeremy M; Hall, Meghan; Kittle, Gale; Berry, James D; Babu, Suma; Andrews, Jinsy; Dagostino, Derek; Tustison, Eric; Giacomelli, Elisa; Scirocco, Erica; Alameda, Gustavo; Locatelli, Eduardo; Ho, Doreen; Quick, Adam; Katz, Jonathan; Heitzman, Daragh; Appel, Stanley H; Shroff, Sheetal; Felice, Kevin; Maragakis, Nicholas J; Simmons, Zachary; Miller, Timothy M; Olney, Nicholas; Weiss, Michael D; Goutman, Stephen A; Fernandes, Joseph Americo; Jawdat, Omar; Owegi, Margaret Ayo; Foster, Laura A; Vu, Tuan; Ilieva, Hristelina; Newman, Daniel S; Arcila-Londono, Ximena; Jackson, Carlayne E; Ladha, Shafeeq; Heiman-Patterson, Terry; Caress, James B; Swenson, Andrea; Peltier, Amanda; Lewis, Richard; Fee, Dominic; Elliott, Matthew; Bedlack, Richard; Kasarskis, Edward J; Elman, Lauren; Rosenfeld, Jeffrey; Walk, David; McIlduff, Courtney; Twydell, Paul; Young, Eufrosina; Johnson, Kristin; Rezania, Kourosh; Goyal, Namita A; Cohen, Jeffrey A; Benatar, Michael; Jones, Vovanti; Glass, Jonathan; Shah, Jaimin; Beydoun, Said R; Wymer, James P; Zilliox, Lindsay; Nayar, Shakti; Pattee, Gary L; Martinez-Thompson, Jennifer; Harvey, Brittany; Patel, Shital; Mahoney, Paul; Duda, Petra W; Cudkowicz, Merit E