杜氏肌营养不良
医学
肌营养不良
遗传增强
肌营养不良蛋白
腺相关病毒
载体(分子生物学)
基因
内科学
遗传学
生物
重组DNA
标识
DOI:10.58347/tml.2023.1686d
摘要
Delandistrogene moxeparvovec-rokl (Elevidys – Sarepta), an adeno-associated virus (AAV) vector-based gene therapy, has received accelerated approval from the FDA for treatment of ambulatory children 4-5 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the dystrophin gene. It is the first gene therapy to be approved in the US for treatment of DMD. Accelerated approval was based on expression of microdystrophin in skeletal muscle, a surrogate endpoint.
科研通智能强力驱动
Strongly Powered by AbleSci AI