Goal 2: Reduce Human Disease

Clinical Trials in Pediatric Sleep Disorders

Effectiveness of medications for cataplexy in children.

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Compelling Question (CQ)

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Pediatric sleep experts agree that there appears to be a mini-epidemic of narcolepsy in children currently, characterized by acute onset of disease with severe symptoms and difficult to control cataplexy, in very young children. No drugs are approved for treatment of cataplexy in children, and drugs typically used in adults are not FDA-approved for use in children. A multicenter trial would be needed in order to accumulate sufficient cases for a randomized controlled trial.

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Idea No. 1533