Lessons from a negative gene therapy trial for Duchenne muscular dystrophy

IF 58.7 1区医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY

Nature Medicine Pub Date : 2024-10-09 DOI:10.1038/s41591-024-03316-9

Simone Spuler, Gian Domenico Borasio, Ulrike Grittner

引用次数: 0

Abstract

Delandistrogene moxeparvovec received FDA approval after a negative trial, which highlights the many complexities and challenges of drug development in this setting.

查看原文本刊更多论文

杜氏肌营养不良症基因疗法试验失败的教训

Delandistrogene moxeparvovec 在试验失败后获得了美国食品及药物管理局（FDA）的批准，这凸显了在这种情况下药物开发的诸多复杂性和挑战。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Nature Medicine 医学-生化与分子生物学

CiteScore

100.90

自引率

0.70%

发文量

525

审稿时长

1 months

期刊介绍： Nature Medicine is a monthly journal publishing original peer-reviewed research in all areas of medicine. The publication focuses on originality, timeliness, interdisciplinary interest, and the impact on improving human health. In addition to research articles, Nature Medicine also publishes commissioned content such as News, Reviews, and Perspectives. This content aims to provide context for the latest advances in translational and clinical research, reaching a wide audience of M.D. and Ph.D. readers. All editorial decisions for the journal are made by a team of full-time professional editors. Nature Medicine consider all types of clinical research, including: -Case-reports and small case series -Clinical trials, whether phase 1, 2, 3 or 4 -Observational studies -Meta-analyses -Biomarker studies -Public and global health studies Nature Medicine is also committed to facilitating communication between translational and clinical researchers. As such, we consider “hybrid” studies with preclinical and translational findings reported alongside data from clinical studies.