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

Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial

Delandistrogene Moxeparvovec 治疗可活动杜氏肌营养不良症患者的两年疗效:3 期 EMBARK 试验

Mendell, Jerry R; Muntoni, Francesco; McDonald, Craig M; Mercuri, Eugenio M; Ciafaloni, Emma; Komaki, Hirofumi; Leon-Astudillo, Carmen; Nascimento, Andrés; Proud, Crystal; Schara-Schmidt, Ulrike; Veerapandiyan, Aravindhan; Zaidman, Craig M; Furgerson, Matthew; Ding, Kai; Singh, Preeti; Potter, Rachael; Asher, Damon R; Murphy, Alexander P; Reid, Carol; Hooper, Gregory; Torre, Carmen O; Manfrini, Marianna; Rodino-Klapac, Louise R

AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.

AAV基因疗法治疗杜氏肌营养不良症:EMBARK 3期随机试验

Mendell Jerry R, Muntoni Francesco, McDonald Craig M, Mercuri Eugenio M, Ciafaloni Emma, Komaki Hirofumi, Leon-Astudillo Carmen, Nascimento Andrés, Proud Crystal, Schara-Schmidt Ulrike, Veerapandiyan Aravindhan, Zaidman Craig M, Guridi Maitea, Murphy Alexander P, Reid Carol, Wandel Christoph, Asher Damon R, Darton Eddie, Mason Stefanie, Potter Rachael A, Singh Teji, Zhang Wenfei, Fontoura Paulo, Elkins Jacob S, Rodino-Klapac Louise R

Quantitative Muscle Magnetic Resonance Outcomes in Patients With Duchenne Muscular Dystrophy: An Exploratory Analysis From the EMBARK Randomized Clinical Trial

杜氏肌营养不良症患者的定量肌肉磁共振结果:来自 EMBARK 随机临床试验的探索性分析

Vandenborne, Krista; Walter, Glenn A; Straub, Volker; Willcocks, Rebecca J; Forbes, Sean C; Mercuri, Eugenio M; Muntoni, Francesco; Ding, Kai; Ennamuri, Sravya; Reid, Carol; Murphy, Alexander P; Manfrini, Marianna; Mendell, Jerry R; Elkins, Jacob S; Rodino-Klapac, Louise R

High-Density Lipoprotein-Associated Cholesterol Abnormalities in a Clinical Outcomes Study of Dysferlin-Deficient Limb-Girdle Muscular Dystrophy Type R2

肌营养不良蛋白缺乏型肢带型肌营养不良症R2型临床结局研究中高密度脂蛋白相关胆固醇异常

White, Zoe; Rufibach, Laura; Dressman, Heather Gordish; Hilsden, Heather; Cox, Dan; Spuler, Simone; Day, John W; Jones, Kristi J; Bharucha-Goebel, Diana X; Salort-Campana, Emmanuelle; Pestronk, Alan; Walter, Maggie C; Paradas, Carmen; Stojkovic, Tanya; Mori-Yoshimura, Madoka; Bravver, Elena; Diaz-Manera, Jordi; Pegoraro, Elena; Mendell, Jerry R; Straub, Volker; Bernatchez, Pascal

Modeling of Dysferlinopathy (LGMDR2) Progression: A Longitudinal Fat Fraction Analysis

肌营养不良蛋白病(LGMDR2)进展建模:纵向脂肪分数分析

Bolano-Diaz, Carla Florencia; Reyngoudt, Harmen; Wilson, Ian J; James, Meredith K; Smith, Fiona Elizabeth; Caldas de Almeida Araujo, Ericky; Gordish-Dressman, Heather; Hilsden, Heather; Rufibach, Laura E; Wallace, Dorothy; Ward, Louise; Stramare, Roberto; Rampado, Alessandro; Smith, Mark; Boisserie, Jean-Marc; Le Louer, Julien; Foster, Sheryl; Peduto, Anthony; Sato, Noriko; Tamaru, Takeshi; Sawyer, Anne Marie; Day, John W; Jones, Kristi J; Walter, Maggie Christine; Stojkovic, Tanya; Mori-Yoshimura, Madoka; Mendell, Jerry R; Pegoraro, Elena; Straub, Volker; Blamire, Andrew M; Carlier, Pierre; Diaz-Manera, Jordi

Limb-Girdle Muscular Dystrophy Scientific Workshop: A Multistakeholder Discussion Focused on Charting the Path Forward for Drug Development

肢带型肌营养不良症科学研讨会:聚焦药物研发未来发展路径的多方利益相关者讨论会

Wicklund, Matthew P; Alfano, Lindsay N; Johnson, Nicholas E; Kang, Peter B; Marks, Peter; Mathews, Katherine D; Mendell, Jerry R; Rodino-Klapac, Louise; Sproule, Douglas; Verdun, Nicole; Bryant, Kathryn

Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74

利用血浆置换术降低对 AAVrh74 具有预先免疫力的非人灵长类动物体内的抗 AAV 抗体

Potter, Rachael A; Peterson, Ellyn L; Griffin, Danielle; Cooper Olson, Grace; Lewis, Sarah; Cochran, Kyle; Mendell, Jerry R; Rodino-Klapac, Louise R

Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy

远程实时视频评估北极星步行评估在杜氏肌营养不良症患者中的有效性

P Lowes, Linda; Alfano, Lindsay N; Iammarino, Megan A; Reash, Natalie F; Giblin, Kathryn; Hu, Larry; Yu, Lixi; Wang, Shufang; Salazar, Rachel; Mendell, Jerry R

Evolving Therapeutic Options for the Treatment of Duchenne Muscular Dystrophy

杜氏肌营养不良症治疗方案的演变

D'Ambrosio, Eleonora S; Mendell, Jerry R

Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy

美国脊髓性肌萎缩症患者腺相关病毒9型抗体血清阳性率

Day, John W; Mendell, Jerry R; Burghes, Arthur H M; van Olden, Rudolf W; Adhikary, Rishi R; Dilly, Keith W