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What's Happening in PWS Research [2020 CONFERENCE VIDEO]

In this conference video, the FPWR Research Team shares highlights from the work supported by the Foundation for Prader-Willi Research.

Join Dr. Theresa Strong and the FPWR Research Team to learn more about what is happening in PWS research. In this 68-minute video, the FPWR Research Team members summarize highlights, recent findings and the latest on PWS research and available resources for PWS families. This is followed by questions from the audience at the 2020 FPWR Virtual Conference.

Click below to watch the video. If you're short on time, scroll down for time stamps to find the portions you're most interested in.


Presentation Summary With Timestamps

1:15 Introduction by FPWR Executive Director Susan Hedstrom. 

FPWR Research and Director Theresa Strong, presenting

  • 2020 has been a year in which some PWS clinical studies have been negatively impacted.
  • However, data mining, analyzing and three Phase 3 trials in PWS were completed.
  • PWS Research Symposium included 20 oral presentations and 23 poster presentations.
  • PWS Research Highlights on Discovery Science, Therapeutics and Genetic Therapy.

7:46 Overview of PWS Research

  • Clinical Trials: Three PWS clinical trials are underway.
  • Highlights of PWS Research – Genetic Therapy
  • The Drug discovery and development pathway and steps to complete an FDA drug approval

10:30 Caroline Verona-Diaz, FPWR Research Team member, presenting on the PWS Clinical Network

  • The three main objectives of Clinical Network for Care of PWS are to establish evidence-based standards of care, improve access for people with PWS to easily become more involved in clinical research and to foster collaborations between clinicians and clinical researchers.
  • Takeaways from the first PWS Clinical Network meeting are to improve clinical care, improve evidence-based decision making based on a central repository for clinical data with the involvement of families, patients and caregivers.

13:25 Jessica Bohonowych, FPWR Associated Director of Research Programs, presenting on the Global PWS Registry

  • One of the main goals of the Global PWS Registry is to act as a comprehensive database of individuals with PWS.
  • The idea of a patchwork quilt to better understand the full spectrum of PWS characteristics.
  • When Clinical Trial sponsors are looking for a set of characteristics on PWS individuals, the Registry can send families with those characteristics the researchers’ contact information so that they can decide whether to follow up with them and participate in the study.

16:04 More highlighted findings from PWS Registry

  • Highlighted data, PWS Registry infographics
  • Collaborations and projects: Developing new endpoints for Clinical Trials, Understanding and Developing Standards of Care in PWS, and Advancing the Understanding of Serious Medical Events in PWS
  • Resources, how to participate on the PWS Registry and contact information.

20:00 Lisa Matesevac, PATH for PWS coordinator, presenting on PATH for PWS

  • As a coordinator, Lisa has been participating in the natural history study that began in October 2018. 
  • Initially, it aimed to have 500 participants, but it exceeded that goal by 40%. It is currently not open for enrollment.
  • Its main goal is to evaluate changes in PWS behaviors and advance understanding of medical history over time in different individuals of different age ranges.
  • There is a high level of compliance with participants completing the required surveys, providing rich data that can help care providers and caregivers.

23:20 PATH for PWS takeaways

  • 20% of PATH participants have had a serious medical event, half of which have occured in the 18+ age group.
  • Medical Events increase with age, and they range from GI issues, mental health issues, blood clots, seizures and surgeries.

24:15 Lauren Schwarz-Roth, FPWR Behavior and Mental Health Programs, presenting on the PWS Mental Health Guidebook

  • Clinical psychologist at the University of Washington
  • Part of the FPWR Research Team, overseeing behavior and mental health research programs at FPWR.
  • Mental Health issues and early signs of challenges have a big impact on PWS families.
  • The PWS Mental Health Guidebook - challenges and benefits of medications for mental health issues and different diagnoses.

31:07 Mindfulness Study is currently recruiting

  • Aimed towards individuals with PWS aged 13-30 years old who display at least one temper outbursts per week and their caregivers.
  • Remotely conducted study, via phone, email and Skype.

32:40 Medication studies that are related to Mental Health Issues which are underway.

  • Guanfacine is an FDA-approved medication for ADHD, which is used clinically to treat temper outbursts, agitation and skin picking. 
  • CBD-V is being studied in individuals with PWS to start the recruitment stage soon.

34:43 Lauren Schwarz will address Social Challenges and tools to address social skill and cognition challenges in PWS individuals in the PWS Fireside Chat (separate 2020 conference presentation)

36:45 Dr. Theresa Strong wraps up by mentioning the importance of participation in FPWR Research and by encouraging families to keep informed about Clinical Trials, upcoming studies and advancements in Research Findings.

38:41 Q&A with all panelists

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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.