The New England journal of medicine
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Randomized Controlled Trial
Oral Orexin Receptor 2 Agonist in Narcolepsy Type 1.
Narcolepsy type 1 is caused by severe loss or lack of brain orexin neuropeptides. ⋯ In a phase 2 trial involving patients with narcolepsy type 1, an orexin receptor 2 agonist resulted in greater improvements on measures of sleepiness and cataplexy than placebo over a period of 8 weeks but was associated with hepatotoxic effects. (Funded by Takeda Development Center Americas; TAK-994-1501 and TAK-994-1504 ClinicalTrials.gov numbers, NCT04096560 and NCT04820842.).