美国食品和药物管理局批准用于治疗杜氏肌营养不良症的 delandistrogene moxeparvovec-rokl：对证据和监管程序的批判性审查。

IF 3.6 3区医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY

Expert Opinion on Biological Therapy Pub Date : 2024-09-01 Epub Date: 2024-08-20 DOI:10.1080/14712598.2024.2394477

Mehul Bhattacharyya, Larry E Miller, Anna L Miller, Ruemon Bhattacharyya

引用次数: 0

摘要

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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The FDA approval of delandistrogene moxeparvovec-rokl for Duchenne muscular dystrophy: a critical examination of the evidence and regulatory process.

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来源期刊

Expert Opinion on Biological Therapy 医学-生物工程与应用微生物

CiteScore

8.60

自引率

0.00%

发文量

审稿时长

3-8 weeks

期刊介绍： Expert Opinion on Biological Therapy (1471-2598; 1744-7682) is a MEDLINE-indexed, international journal publishing peer-reviewed research across all aspects of biological therapy. Each article is structured to incorporate the author’s own expert opinion on the impact of the topic on research and clinical practice and the scope for future development. The audience consists of scientists and managers in the healthcare and biopharmaceutical industries and others closely involved in the development and application of biological therapies for the treatment of human disease. The journal welcomes: Reviews covering therapeutic antibodies and vaccines, peptides and proteins, gene therapies and gene transfer technologies, cell-based therapies and regenerative medicine Drug evaluations reviewing the clinical data on a particular biological agent Original research papers reporting the results of clinical investigations on biological agents and biotherapeutic-based studies with a strong link to clinical practice Comprehensive coverage in each review is complemented by the unique Expert Collection format and includes the following sections: Expert Opinion – a personal view of the data presented in the article, a discussion on the developments that are likely to be important in the future, and the avenues of research likely to become exciting as further studies yield more detailed results; Article Highlights – an executive summary of the author’s most critical points.