NT2 shares all symptoms of NT1, except cataplexy. NT1 has a unique pathophysiology: it is due to the irreversible destruction of hypocretin/orexin (ORX) neurons with an absence or low levels of ORX in the cerebrospinal fluid (CSF), probably by an autoimmune mechanism, in genetically predisposed individuals. mood disorders, anxiety, eating disorders and attention-deficit hyperactivity disorder), and other sleep disorders such as restless legs syndrome, 6 periodic leg movements, 7 rapid eye movement (REM) and non-REM (NREM) sleep parasomnias. obesity, type 2 diabetes, sleep apnea, cardiovascular diseases), 4, 5 neuropsychiatric comorbidities (e.g. Patients often present several comorbid conditions including cardiometabolic comorbidities (e.g. 3 It often starts in childhood or adolescence and requires a lifelong treatment. This rare disease affects 0.026–0.05% of the population. 1, 2 Patients may also exhibit sleep paralysis, hypnagogic and hypnopompic hallucinations, and disturbed nocturnal sleep. NT1 is a chronic and disabling sleep disorder with excessive daytime sleepiness (EDS) and cataplexy, sudden loss of muscle tone triggered by positive emotions, being the main symptoms of the disorder. Given the different clinical, biological and genetic profiles, narcolepsy may provide a nice example for developing personalized medicine in orphan diseases, that could ultimately aid in similar research and clinical efforts for other conditions. Further well-designed controlled trials are required to determine if they could really impact on the natural history of the disease. In addition, immune-based therapies administered as early as possible after disease onset could theoretically slow down or stop the destruction of ORX neurons in some selected patients. The discovery of ORX deficiency in NT1 opened new therapeutic options oriented towards ORX-based therapies, especially nonpeptide ORX receptor agonists that are currently under development. Sodium oxybate, used for decades in adult patients with narcolepsy, was recently shown to be effective and safe in childhood narcolepsy. Solriamfetol, a phenylalanine derivative with dopaminergic and noradrenergic activity will be soon a new therapeutic option to treat EDS in NT1 and NT2. Pitolisant, a selective histamine H3 receptor inverse agonist, has been recently approved to treat patients with NT1 and NT2. Narcolepsy is an active area for drug development, and new wake-promoting agents have been developed over the past years. The main and most disabling symptom, EDS, is managed with psychostimulants, as modafinil/armodafinil, methylphenidate, or amphetamines as a third-line therapy. Despite major advances in our understanding of the neurobiological basis of NT1, management remains nowadays only symptomatic. Little is known about NT2 etiology and prevalence, sharing with NT1 excessive daytime sleepiness (EDS) and dysregulation of rapid eye movement (REM) sleep, but without cataplexy and loss of ORX neurons. Narcolepsy type 1 (NT1) is a chronic orphan disorder, caused by the selective and irreversible loss of hypocretin/orexin (ORX) neurons, by a probable autoimmune process. All subjects Allied Health Cardiology & Cardiovascular Medicine Dentistry Emergency Medicine & Critical Care Endocrinology & Metabolism Environmental Science General Medicine Geriatrics Infectious Diseases Medico-legal Neurology Nursing Nutrition Obstetrics & Gynecology Oncology Orthopaedics & Sports Medicine Otolaryngology Palliative Medicine & Chronic Care Pediatrics Pharmacology & Toxicology Psychiatry & Psychology Public Health Pulmonary & Respiratory Medicine Radiology Research Methods & Evaluation Rheumatology Surgery Tropical Medicine Veterinary Medicine Cell Biology Clinical Biochemistry Environmental Science Life Sciences Neuroscience Pharmacology & Toxicology Biomedical Engineering Engineering & Computing Environmental Engineering Materials Science Anthropology & Archaeology Communication & Media Studies Criminology & Criminal Justice Cultural Studies Economics & Development Education Environmental Studies Ethnic Studies Family Studies Gender Studies Geography Gerontology & Aging Group Studies History Information Science Interpersonal Violence Language & Linguistics Law Management & Organization Studies Marketing & Hospitality Music Peace Studies & Conflict Resolution Philosophy Politics & International Relations Psychoanalysis Psychology & Counseling Public Administration Regional Studies Religion Research Methods & Evaluation Science & Society Studies Social Work & Social Policy Sociology Special Education Urban Studies & Planning BROWSE JOURNALS
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