Disease management of Prader-Willi syndrome

Research output: Contribution to journalReview articlepeer-review

13 Scopus citations


Prader-Willi Syndrome (PWS), first described in 1956, is a unique genetic condition with a prevalence of 1 in 10,000 - 25,000. Features include severe life-long hypotonia, insatiable appetite, short stature, obsessive-compulsive behaviour, morbid obesity, hypogonadism, kyphosis, scoliosis and osteoporosis. Studies beginning in the 1970s demonstrated that PWS is associated with a deficiency in growth hormone. Growth hormone treatment in children with PWS improves linear growth and more importantly leads to an increased muscle mass, bone mineral density and physical performance. In mid-2000, growth hormone became the first pharmaceutical approved in the US and Europe for the treatment of childhood PWS. It is now considered an essential part of comprehensive care for this condition. Ongoing studies address issues of growth hormone dosage, long-term efficacy, effects on neonatal and childhood growth and development and effects in adults with PWS.

Original languageEnglish (US)
Pages (from-to)1451-1459
Number of pages9
JournalExpert Opinion on Pharmacotherapy
Issue number10
StatePublished - Oct 1 2002
Externally publishedYes


  • Body composition
  • Growth hormone
  • Hyperphagia
  • Insulin-like growth factor
  • Prader-Willi syndrome
  • obesity

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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