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Nusinersen, an anitisense oligonucleotide, has been recently approved by the US FDA for treatment of SMA ( Huntington Disease Huntington disease (HD) is a rare genetic autosomal dominant neurodegenerative syndrome triggered by expanded CAG repeats in the huntingtin gene, which leads to pathological elongation of huntingtin with a polyglutamine tract

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Narcolepsy is characterized by disruption of that architecture, particularly in the transition between wakefulness and REM sleep