The hope of an early trajectory-modifying treatment
The following teams are driving this innovative therapeutic approach:
• The team at the Rare Diseases Reference Centre for Prader-Willi Syndrome and other rare forms of obesity with eating disorders, led by Professor Maithé Tauber at the Toulouse University Hospital, for clinical studies,
• The research team led by Françoise Muscatelli, Research Director of the CNRS (French National Centre for Scientific Research) at INSERM’s Institut de Neurobiologie de la Méditerranée (Marseilles), Unit 901, for pre-clinical studies.
It has been shown that people with Prader-Willi syndrome have a reduced number of oxytocin-producing neurons in the hypothalamus, leading to oxytocin deficiency, while other hormones and neurohormones are not impaired (Swaab 1995). These data have been confirmed by PW-like animal models – Necdin (Muscatelli 2000) and Magel2 (Schaller 2010) – which showed an alteration in the maturation of the oxytocinergic system from birth.