Emerging treatments
Mazindol
Mazindol, a norepinephrine and dopamine reuptake inhibitor, is a wake-promoting agent that has received orphan drug designation from the Food and Drug Administration for the treatment of narcolepsy.
Hypocretin peptide supplementation
Hypocretin peptide supplementation or cell replacement therapies seem to be a rational approach in the treatment of narcolepsy type 1; nevertheless, the studies are still in early phases and explore different routes of administration.[113] In the future, it may become possible to transplant hypocretin-producing cells in the brain or use gene therapy to treat hypocretin deficiency in patients with narcolepsy.
Baclofen
Baclofen, a gamma-aminobutyric acid-B agonist, improved total sleep time and delta waves during sleep, but not cataplexy or excessive daytime sleepiness, in a small study of teenagers with recent-onset narcolepsy-cataplexy syndrome.[114] Subsequent case studies, however, suggest that baclofen may be of benefit in patients with narcolepsy (who had an inadequate response or experienced adverse effects to initial therapies) with multiple daily cataplexy episodes or severe excessive daytime sleepiness.[115][116]
Other experimental therapies
Other experimental treatments include slow-wave sleep enhancers (e.g., pregabalin, gabapentin), intravenous gamma globulins, tramadol, L-carnitine, and corticosteroids.[117][118] Future therapeutic targets for cataplexy are oriented toward immune-based therapies at early stages of the disease and hypocretin replacement therapy.
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