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

Assessment of safety and efficacy of risdiplam treatment in adults with spinal muscular atrophy

利司帕林治疗成人脊髓性肌萎缩症的安全性和有效性评估

Jaworek, Andrea; Jira, Kathryn; Allen, Matti; Zhao, Songzhu; Kelly, Kristina; Moravec, Trevor; Tellez, Marco; Heintzman, Sarah; Reynolds, Jerold; Sterling, Gary; Kolb, Stephen J; Arnold, William David; Elsheikh, Bakri

Machine learning-enhanced electrical impedance myography to diagnose and track spinal muscular atrophy progression

利用机器学习增强的电阻抗肌电图诊断和追踪脊髓性肌萎缩症的进展

Sonbas Cobb, Buket; Kolb, Stephen J; Rutkove, Seward B

Evidence-based consensus guidelines for ALS genetic testing and counseling

基于证据的ALS基因检测和咨询共识指南

Roggenbuck, Jennifer; Eubank, Breda H F; Wright, Joshua; Harms, Matthew B; Kolb, Stephen J

Identifying Biomarkers of Spinal Muscular Atrophy for Further Development

识别脊髓性肌萎缩症的生物标志物以促进其进一步发展

Glascock, Jacqueline; Darras, Basil T; Crawford, Thomas O; Sumner, Charlotte J; Kolb, Stephen J; DiDonato, Christine; Elsheikh, Bakri; Howell, Kelly; Farwell, Wildon; Valente, Marta; Petrillo, Marco; Tingey, Jessica; Jarecki, Jill

A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALS

一项针对肌萎缩侧索硬化症(ALS)患者的瑞德西姆替夫(Reldesemtiv)II期双盲随机剂量范围试验。

Shefner, Jeremy M; Andrews, Jinsy A; Genge, Angela; Jackson, Carlayne; Lechtzin, Noah; Miller, Timothy M; Cockroft, Bettina M; Meng, Lisa; Wei, Jenny; Wolff, Andrew A; Malik, Fady I; Bodkin, Cynthia; Brooks, Benjamin R; Caress, James; Dionne, Annie; Fee, Dominic; Goutman, Stephen A; Goyal, Namita A; Hardiman, Orla; Hayat, Ghazala; Heiman-Patterson, Terry; Heitzman, Daragh; Henderson, Robert D; Johnston, Wendy; Karam, Chafic; Kiernan, Matthew C; Kolb, Stephen J; Korngut, Lawrence; Ladha, Shafeeq; Matte, Genevieve; Mora, Jesus S; Needham, Merrilee; Oskarsson, Bjorn; Pattee, Gary L; Pioro, Erik P; Pulley, Michael; Quan, Dianna; Rezania, Kourosh; Schellenberg, Kerri L; Schultz, David; Shoesmith, Christen; Simmons, Zachary; Statland, Jeffrey; Sultan, Shumaila; Swenson, Andrea; Berg, Leonard H Van Den; Vu, Tuan; Vucic, Steve; Weiss, Michael; Whyte-Rayson, Ashley; Wymer, James; Zinman, Lorne; Rudnicki, Stacy A

Update on Biomarkers in Spinal Muscular Atrophy

脊髓性肌萎缩症生物标志物研究进展

Pino, Megan G; Rich, Kelly A; Kolb, Stephen J

Persistent neuromuscular junction transmission defects in adults with spinal muscular atrophy treated with nusinersen

接受诺西那生治疗的脊髓性肌萎缩症成年患者持续存在神经肌肉接头传递缺陷

Arnold, W David; Severyn, Steven; Zhao, Songzhu; Kline, David; Linsenmayer, Matthew; Kelly, Kristina; Tellez, Marco; Bartlett, Amy; Heintzman, Sarah; Reynolds, Jerry; Sterling, Gary; Weaver, Tristan; Rajneesh, Kiran; Burghes, Arthur H M; Kolb, Stephen J; Elsheikh, Bakri

Seven-Year Experience From the National Institute of Neurological Disorders and Stroke-Supported Network for Excellence in Neuroscience Clinical Trials

来自美国国家神经疾病与卒中研究所支持的神经科学临床试验卓越网络的七年经验

Cudkowicz, Merit; Chase, Marianne K; Coffey, Christopher S; Ecklund, Dixie J; Thornell, Brenda J; Lungu, Codrin; Mahoney, Katy; Gutmann, Laurie; Shefner, Jeremy M; Staley, Kevin J; Bosch, Michael; Foster, Eric; Long, Jeffrey D; Bayman, Emine O; Torner, James; Yankey, Jon; Peters, Richard; Huff, Trevis; Conwit, Robin A; Shinnar, Shlomo; Patch, Donna; Darras, Basil T; Ellis, Audrey; Packer, Roger J; Marder, Karen S; Chiriboga, Claudia A; Henchcliffe, Claire; Moran, Joyce Ann; Nikolov, Blagovest; Factor, Stewart A; Seeley, Carole; Greenberg, Steven M; Amato, Anthony A; DeGregorio, Sara; Simuni, Tanya; Ward, Tina; Kissel, John T; Kolb, Stephen J; Bartlett, Amy; Quinn, Joseph F; Keith, Kellie; Levine, Steven R; Gilles, Nadege; Coyle, Patricia K; Lamb, Jessica; Wolfe, Gil I; Crumlish, Annemarie; Mejico, Luis; Iqbal, Muhammad Maaz; Bowen, James D; Tongco, Caryl; Nabors, Louis B; Bashir, Khurram; Benge, Melanie; McDonald, Craig M; Henricson, Erik K; Oskarsson, Björn; Dobkin, Bruce H; Canamar, Catherine; Glauser, Tracy A; Woo, Daniel; Molloy, Angela; Clark, Peggy; Vollmer, Timothy L; Stein, Alexander J; Barohn, Richard J; Dimachkie, Mazen M; Le Pichon, Jean-Baptiste; Benatar, Michael G; Steele, Julie; Wechsler, Lawrence; Clemens, Paula R; Amity, Christine; Holloway, Robert G; Annis, Christine; Goldberg, Mark P; Andersen, Mariam; Iannaccone, Susan T; Smith, A Gordon; Singleton, J Robinson; Doudova, Mariana; Haley, E Clarke; Quigg, Mark S; Lowenhaupt, Stephanie; Malow, Beth A; Adkins, Karen; Clifford, David B; Teshome, Mengesha A; Connolly, Noreen

Incidence of pathogenic, likely pathogenic, and uncertain ALS variants in a clinic cohort

临床队列中致病性、可能致病性和不确定性ALS变异的发生率

Roggenbuck, Jennifer; Palettas, Marilly; Vicini, Leah; Patel, Radha; Quick, Adam; Kolb, Stephen J

Longitudinal natural history of type I spinal muscular atrophy: a critical review

I型脊髓性肌萎缩症的纵向自然史:一项批判性综述

Mercuri, Eugenio; Lucibello, Simona; Perulli, Marco; Coratti, Giorgia; de Sanctis, Roberto; Pera, Maria Carmela; Pane, Marika; Montes, Jacqueline; de Vivo, Darryl C; Darras, Basil T; Kolb, Stephen J; Finkel, Richard S