On Tuesday June 15th at 17:00 – 18:00 CET an Endo-ERN webinar about “Clinical trials with new drugs in PWS” will be given by prof. Maithé Tauber from CHU Toulouse, France.
Prader-Willi syndrome is a rare genetic neurodevelopmental disorder that results from the loss of gene expression of paternally inherited maternally imprinted genes of chromosome 15 at positions q11- q13. It is now admitted that the phenotype is explained by the impaired hypothalamic development and function which drives the developmental trajectory including nutritional, behavioural, endocrine and metabolic aspects. The nutritional trajectory is very specific with a paradoxical sequence that starts with poor feeding with failure to thrive and anorexia, and further switches to unexplained excessive weight gain followed by hyperphagia leading to early severe obesity. In 2000, GH has been approved by the European Medicines Agency (EMA) for treatment of children with PWS and is the only treatment approved in PWS. To date, there is no marketed treatment targeting feeding and behaviour problems that represent the major unmet clinical needs. Several clinical trials with various drugs targeting hyperphagia and obesity have been implemented in PWS recently. The webinar will focus on the clinical trials and discuss the outcome of the studies and safety.