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

Evidentiary basis of the first regulatory qualification of a digital primary efficacy endpoint

数字主要疗效终点首次监管资格认定的证据基础

Servais, Laurent; Strijbos, Paul; Poleur, Margaux; Mirea, Andrada; Butoianu, Nina; Sansone, Valeria A; Vuillerot, Carole; Schara-Schmidt, Ulrike; Scoto, Mariacristina; Seferian, Andreea M; Previtali, Stefano C; Tulinius, Már; Nascimento, Andrés; Furlong, Pat; Singh, Teji; Dreghici, Roxana Donisa; Goemans, Nathalie; Mercuri, Eugenio; Straub, Volker; Ormazabal, Maitea Guridi; Braid, Jessica; Muntoni, Francesco; Tricot, Alexis; Annoussamy, Mélanie; Eggenspieler, Damien

Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy

在接受 delandistrogene moxeparvovec 基因治疗的杜氏肌营养不良症患者中,SRP-9001 肌营养不良蛋白的表达和运动功能的稳定可持续长达 2 年。

Mendell, Jerry R; Shieh, Perry B; McDonald, Craig M; Sahenk, Zarife; Lehman, Kelly J; Lowes, Linda P; Reash, Natalie F; Iammarino, Megan A; Alfano, Lindsay N; Sabo, Brenna; Woods, Jeremy D; Skura, Christy L; Mao, Howard C; Staudt, Loretta A; Griffin, Danielle A; Lewis, Sarah; Wang, Shufang; Potter, Rachael A; Singh, Teji; Rodino-Klapac, Louise R