From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD1 Mutated Amyotrophic Lateral Sclerosis.

IF 4 2区医学 Q2 BIOCHEMISTRY & MOLECULAR BIOLOGY

Nucleic acid therapeutics Pub Date : 2023-08-01 DOI:10.1089/nat.2023.0027

Willeke van Roon-Mom, Chantal Ferguson, Annemieke Aartsma-Rus

引用次数: 2

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从未能达到临床终点到美国食品和药物管理局批准:第15个反义寡核苷酸疗法Qalsody (Tofersen)被批准用于治疗SOD1突变的肌萎缩侧索硬化症。

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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来源期刊

Nucleic acid therapeutics BIOCHEMISTRY & MOLECULAR BIOLOGY-CHEMISTRY, MEDICINAL

CiteScore

7.60

自引率

7.50%

发文量

审稿时长

>12 weeks

期刊介绍： Nucleic Acid Therapeutics is the leading journal in its field focusing on cutting-edge basic research, therapeutic applications, and drug development using nucleic acids or related compounds to alter gene expression. The Journal examines many new approaches for using nucleic acids as therapeutic agents or in modifying nucleic acids for therapeutic purposes including: oligonucleotides, gene modification, aptamers, RNA nanoparticles, and ribozymes.