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

The Muscular Dystrophy Association's neuroMuscular ObserVational Research Data Hub (MOVR): Design, Methods, and Initial Observations

肌肉萎缩症协会的神经肌肉观察研究数据中心(MOVR):设计、方法和初步观察

Kilroy, Elisabeth A; Burris, Rachael; Javelosa, Edritz; Waits, Jessica; Lek, Angela; Rodgers, Rayne; Opgenorth, Hayley; Hesterlee, Sharon

Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents

基因疗法作为杜氏肌营养不良症的潜在治疗选择:一项针对患者及其父母的定性偏好研究

Landrum Peay, Holly; Fischer, Ryan; Tzeng, Janice P; Hesterlee, Sharon E; Morris, Carl; Strong Martin, Amy; Rensch, Colin; Smith, Edward; Ricotti, Valeria; Beaverson, Katherine; Wand, Hannah; Mansfield, Carol

The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development

TREAT-NMD治疗咨询委员会(TACT):一种促进孤儿药研发的创新型风险降低模式

Heslop, Emma; Csimma, Cristina; Straub, Volker; McCall, John; Nagaraju, Kanneboyina; Wagner, Kathryn R; Caizergues, Didier; Korinthenberg, Rudolf; Flanigan, Kevin M; Kaufmann, Petra; McNeil, Elizabeth; Mendell, Jerry; Hesterlee, Sharon; Wells, Dominic J; Bushby, Kate

Patient Engagement Practices in Clinical Research among Patient Groups, Industry, and Academia in the United States: A Survey

美国患者团体、产业界和学术界在临床研究中的患者参与实践:一项调查

Smith, Sophia K; Selig, Wendy; Harker, Matthew; Roberts, Jamie N; Hesterlee, Sharon; Leventhal, David; Klein, Richard; Patrick-Lake, Bray; Abernethy, Amy P

A call for transparent reporting to optimize the predictive value of preclinical research

呼吁提高报告透明度,以优化临床前研究的预测价值

Landis, Story C; Amara, Susan G; Asadullah, Khusru; Austin, Chris P; Blumenstein, Robi; Bradley, Eileen W; Crystal, Ronald G; Darnell, Robert B; Ferrante, Robert J; Fillit, Howard; Finkelstein, Robert; Fisher, Marc; Gendelman, Howard E; Golub, Robert M; Goudreau, John L; Gross, Robert A; Gubitz, Amelie K; Hesterlee, Sharon E; Howells, David W; Huguenard, John; Kelner, Katrina; Koroshetz, Walter; Krainc, Dimitri; Lazic, Stanley E; Levine, Michael S; Macleod, Malcolm R; McCall, John M; Moxley, Richard T 3rd; Narasimhan, Kalyani; Noble, Linda J; Perrin, Steve; Porter, John D; Steward, Oswald; Unger, Ellis; Utz, Ursula; Silberberg, Shai D