Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.

导出 LinkOut

翻译打印收藏纠错

作者： Craig M,McDonald ^[1] ; Perry B,Shieh ^[2] ; Hoda Z,Abdel-Hamid ^[3] ; Anne M,Connolly ^[4] ; Emma,Ciafaloni ^[5] ; Kathryn R,Wagner ^[6] ; Nathalie,Goemans ^[7] ; Eugenio,Mercuri ^[8] ; Navid,Khan ^[9] ; Erica,Koenig ^[10] ; Jyoti,Malhotra ^[10] ; Wenfei,Zhang ^[10] ; Baoguang,Han ^[10] ; Jerry R,Mendell ^[10] ; the Italian DMD Telethon Registry Study Group, Leuven NMRC Registry Investigators, CINRG Duchenne Natural History Investigators, and PROMOVI Trial Clinical Investigators ^[4]

作者单位： Departments of Physical Medicine & Rehabilitation and Pediatrics, University of California Davis Health System and School of Medicine, Sacramento, CA, USA. ^[1] Department of Neurology, University of California, Los Angeles, Los Angeles, CA, USA. ^[2] UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA, USA. ^[3] Pediatrics, Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA. ^[4] University of Rochester Medical Center, Rochester, NY, USA. ^[5] Center for Genetic Muscle Disorders, Kennedy Krieger Institute, Baltimore, MD, USA. ^[6] Departments of Neurology and Neuroscience, Johns Hopkins School of Medicine, Baltimore, MD, USA. ^[7] Department of Pediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium. ^[8] Pediatric Neurology, Catholic University, Rome, Italy. ^[9] Sarepta Therapeutics, Inc., Cambridge, MA, USA. ^[10]

关键词 Muscular dystrophy clinical trial duchenne phase 3 safety treatment efficacy

主题词青少年(Adolescent);儿童(Child);疾病恶化(Disease Progression);肌营养不良蛋白(Dystrophin);外显子(Exons);人类(Humans);男(雄)性(Male);肌营养不良, 杜氏(Muscular Dystrophy, Duchenne);突变(Mutation);肺活量(Vital Capacity)

DOI 10.3233/JND-210643

PMID 34120909

发布时间 2022-02-03

翻译满意度评价：

准确一般字义偏差语意偏离译文作用小提交

浏览6

Journal of neuromuscular diseases

2021年8卷6期

989-1001页

相似文献

中文期刊
外文期刊
学位论文
会议论文

检索历史清除

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.

Journal of neuromuscular diseases

相似文献

Journal of neuromuscular diseases

相关期刊

参考文献：
指定格式：	NoteExpress EndNote RefWorks NoteFirst

检索历史 清除

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.

Journal of neuromuscular diseases

相似文献

Journal of neuromuscular diseases

相关期刊

检索历史清除