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
|00:00:00||The Drug Development Process by Dr. Theresa Strong|
|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)|
|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?