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

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

Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy

来自 EMBARK 的照护者总体印象观察:一项评估 Delandistrogene Moxeparvovec 对可活动杜氏肌营养不良症患者的 3 期研究

McDonald, Craig M; Elkins, Jacob S; Dharmarajan, Sai; Gooch, Katherine; Ciobanu, Teofil; Lansdall, Claire J; Murphy, Alexander P; McDougall, Fiona; Mercuri, Eugenio M; Audhya, Ivana