New Treatment Perspectives in Prader-Willi syndrome

This thesis presents a detailed description of the studies, which were performed to increase the knowledge about Prader-Willi syndrome (PWS) and to improve the care for patients with PWS.
Serum levels of the appetite-regulating hormones acylated and unacylated ghrelin were measured in patients participating in the Dutch PWS Cohort Study, in collaboration with two French PWS centers. These hormone levels were also measured during oral glucose tolerance tests in participants of the Young Adult PWS Study.
In the randomized, double-blinded, placebo-controlled PWS Oxytocin Study, the effects of 4 weeks of intranasally administered oxytocin versus 4 weeks of placebo on social behaviour and eating behaviour were investigated in children with PWS.
In the randomized, double-blinded, placebo-controlled GH study (PWS Transition Study), the study population consisted of young adults with PWS who were GH-treated during childhood and had attained adult height, and thus had to stop GH treatment. In this specific group, we evaluated the effects of 1 year of GH treatment versus 1 year of placebo on body composition, metabolic health profile and cognition.