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- W3167753753 endingPage "884" @default.
- W3167753753 startingPage "869" @default.
- W3167753753 abstract "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 is no treatment, much less cure; therefore, there is an urgent need for new therapies to extend and improve quality of life for persons who suffer from them. Here we focus specifically on rare neuromuscular diseases. Currently, genetic medicines using short antisense oligonucleotides (ASO) or small interfering ribonucleic acids that target RNA transcripts are achieving spectacular success in treating these diseases. For Duchenne muscular dystrophy (DMD), the state-of-the-art is an exon skipping therapy using an antisense oligonucleotide, which is prototypical of advanced precision medicines. Very recently, golodirsen and viltolarsen, for treatment of DMD patients amenable to skipping exon 53, have been approved by regulatory agencies in the USA and Japan, respectively. Here, we review scientific and clinical progress in developing new oligonucleotide therapeutics for selected rare neuromuscular diseases, discussing their efficacy and limitations." @default.
- W3167753753 created "2021-06-22" @default.
- W3167753753 creator A5070566847 @default.
- W3167753753 creator A5071796422 @default.
- W3167753753 date "2021-11-02" @default.
- W3167753753 modified "2023-09-27" @default.
- W3167753753 title "Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases" @default.
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- W3167753753 doi "https://doi.org/10.3233/jnd-200560" @default.
- W3167753753 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/34092651" @default.
- W3167753753 hasPublicationYear "2021" @default.
- W3167753753 type Work @default.