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The Prader-Willi syndrome Profile: validation of a new measure of behavioral and emotional problems in Prader-Willi syndrome.

Elisabeth M DykensElizabeth RoofHailee Hunt-Hawkins
Published in: Orphanet journal of rare diseases (2024)
The PWS Profile is a valid, psychometrically-sound questionnaire that already has shown responsivity to treatment in a previous clinical trial. The Profile can extend the reach of future clinical trials by evaluating the impact of novel agents not only on hyperphagia, but also on the emotional and behavioral problems that characterize PWS.
Keyphrases
  • clinical trial
  • growth hormone
  • mental health
  • case report
  • study protocol
  • open label
  • cross sectional
  • randomized controlled trial
  • phase iii
  • current status
  • psychometric properties
  • patient reported
  • placebo controlled