Emerging oligonucleotide therapeutics for rare neuromuscular diseases

Research and drug development concerning rare diseases are at the cutting edge of scientific technology. To date, over 7,000 rare diseases have been identified. Despite their individual rarity, 1 in 10 individuals worldwide is affected by a rare condition. For the majority of these diseases, there i...

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Main Authors: Aoki, Y, Wood, MJA
格式: Journal article
语言:English
出版: IOS Press 2021