2019 PWS Clinical Trials Panel [VIDEO]

The PWS Clinical Trials Panel presentation in the video below took place at FPWR’s 2019 Annual Research Symposium and Family Conference. The 90-minute session opens with a quick overview of the drug development process and is followed by presentations about five PWS clinical trials. Each panelist was given seven minutes to share information about their ongoing study before answering questions from the moderator. Timestamps are provided below for easy viewing of specific content.

You can view additional 20-minute videos on some of the clinical trials by clicking on the links provided.



Presentation Sections With Timestamps

Time Presentation
00:00:00 The Drug Development Process by Dr. Theresa Strong
00:07:46 Guanfacine
00:19:50 Livoletide (Additional 20-minute webinar available from spring 2019)
00:33:12 Carbetocin (Additional 20-minute webinar available from spring 2019)
00:41:05 Diazoxide (DCCR) (Additional 20-minute webinar available from spring 2019)
00:51:54 Pitolisant
01:09:35 Moderator Questions
01:16:40 Open Q&A from Audience

Questions Asked by the Moderator

When someone participates in a trial, what individual data will be shared with them? What results or values will you give to the patients?

We can expect that one or all of the studies presented today will be completed in 2020. What should we expect after the close of the study? How long might it be before it goes before the FDA and if approved, how long could we expect it to take before the drug is available for prescription?

While waiting for drug approval, will compassionate care be available to allow families who have participated in the clinical trial to remain on the drug?

PWS Clinical Trials

Topics: Research

Susan Hedstrom


Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.