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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Autori principali: Aoki, Y, Wood, MJA
Natura: Journal article
Lingua:English
Pubblicazione: IOS Press 2021