Progress in the Treatment of Antisense Oligonucleotides in Duchenne Muscular Dystrophy

Progress in the Treatment of Antisense Oligonucleotides in Duchenne Muscular Dystrophy

In recent years, antisense oligonucleotide (ASO) has been very active in the field of rare disease research and development, especially in Duchenne muscular dystrophy, where it made a major breakthrough. Duchenne muscular dystrophy (DMD) is a rare childhood myopathy caused by mutations in the dystro...

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Bibliographic Details
Main Authors: XU Tingting, ZUO Wei, LIU Xin, FAN Qianqian, GAO Yang, LIANG Chunsu, ZHANG Bo
Format: Article
Language:zho
Published: Editorial Office of Journal of Rare Diseases 2022-04-01
Series:罕见病研究
Subjects:
antisense oligonucleotides
duchenne muscular dystrophy
rare diseases
pharmacokinetics
efficacy and safety
Online Access:https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2022.02.015

Internet

https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2022.02.015

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