DataSheet1_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.docx

Introduction: Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy designed for targeted expression of SRP-9001 dystrophin protein, a shortened dystrophin retaining key functional domains of the wild-type protein.Methods: This Phase 2, double-blind, two-part (48 weeks per part) crossover study (SRP-9001-102 [Study 102]; NCT03769116) evaluated delandistrogene moxeparvovec in patients, aged ≥4 to

引言：德兰地司妥肌蛋白基因转移疗法（SRP-9001）系一项正在研发的基因治疗技术，旨在实现SRP-9001型肌萎缩蛋白的定向表达。该肌萎缩蛋白为缩短型，保留了野生型蛋白的关键功能域。方法：本研究为第2期双盲、两阶段（每阶段48周）交叉研究（SRP-9001-102[研究102]; NCT03769116），旨在评估德兰地司妥肌蛋白基因转移疗法在年龄≥4岁患者的疗效。