Mission

The mission of the PWS consortium is to accelerate clinical trials for PWS through the establishment of a collaborative, pre-competitive and international consortium that will leverage expertise and perspective of stakeholders from industry, academia, governmental agencies and patient organizations to address unmet scientific, technical, clinical and regulatory needs for clinical trials for PWS. 

Overall objectives

  • Create a multi-disciplinary expert network incorporating the voice of patients, industry and academics to identify key challenges and championing solutions to advance current and future clinical trials for PWS

  • Develop a PWS evidence-based knowledgebase that provides comprehensive information useful for advancing PWS clinical trials

  • Facilitate regulatory decision-making

  • Facilitate sharing of data and bioresources

  • Develop tools and methodologies to improve the clinical trial process for PWS

  • Become a reference for PWS clinical trials

Deliverables

  • Provide recommendations and guidelines to the PWS community

  • Develop tools, standards, platforms broadly available for the entire PWS community

  • Develop and implement new studies to advance the mission of the consortium

The consortium has been joined by: Levo Therapeutics, Millendo Therapeutics, Rhythm Therapeutics, Saniona, Soleno Therapeutics, Zafgen, Inversago, the Foundation for Prader-Willi Research, the Prader-Willi Syndrome Association, the International Prader-Willi Syndrome Organization, the Prader-Willi France Association, and researchers Drs. E. Dykens (Vanderbilt University), S. Einfeld (University of Sydney), L. Rice (University of Sydney), E. Roof (Vanderbilt University), Hailee Hunt (Vanderbilt Kennedy Center), Jessica Duis (Vanderbilt University, School of Medicine), P. Salehi (University of WA, Seattle Children’s Hospital), B. Taylor (Albert Einstein College of Medicine-Montefiore Medical Center), A. Caixas (Autonomous University of Barcelona), C. Höybye (Karolinska Institute), A. Dimitropoulos (Case Western Reserve Univ.), J. Miller (Florida University).

© 2016 PWS Clinical Trial Consortium