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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Hlavní autoři: Aoki, Y, Wood, MJA
Médium: Journal article
Jazyk:English
Vydáno: IOS Press 2021