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- W2795926027 abstract "Narcolepsy with cataplexy is most commonly caused by a loss of hypocretin/orexin peptide-producing neurons in the hypothalamus (i.e., Narcolepsy Type 1). Since hypocretin deficiency is assumed to be the main cause of narcoleptic symptoms, hypocretin replacement will be the most essential treatment for narcolepsy. Unfortunately, this option is still not available clinically. There are many potential approaches to replace hypocretin in the brain for narcolepsy such as intranasal administration of hypocretin peptides, developing small molecule hypocretin receptor agonists, hypocretin neuronal transplantation, transforming hypocretin stem cells into hypothalamic neurons, and hypocretin gene therapy. Together with these options, immunotherapy treatments to prevent hypocretin neuronal death should also be developed.In this review, we overview the pathophysiology of narcolepsy and the current and emerging treatments of narcolepsy especially focusing on hypocretin receptor based treatments.Among hypocretin replacement strategies, developing non-peptide hypocretin receptor agonists is currently the most encouraging since systemic administration of a newly synthesized, selective hypocretin receptor 2 agonist (YNT-185) has been shown to ameliorate symptoms of narcolepsy in murine models. If this option is effective in humans, hypocretin cell transplants or gene therapy technology may become realistic in the future." @default.
- W2795926027 created "2018-04-13" @default.
- W2795926027 creator A5029463010 @default.
- W2795926027 creator A5030128079 @default.
- W2795926027 creator A5056588640 @default.
- W2795926027 creator A5060824249 @default.
- W2795926027 creator A5077524124 @default.
- W2795926027 creator A5079847965 @default.
- W2795926027 date "2018-04-03" @default.
- W2795926027 modified "2023-10-16" @default.
- W2795926027 title "An overview of hypocretin based therapy in narcolepsy" @default.
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