FPWR Blog

This guest blog was contributed by researchers Elisabeth Dykens and Elizabeth Roof of Vanderbilt University 

Does your loved one with PWS have hyperphagia? The answer isn't as simple as yes or no. Even if your loved one does not display the hallmark hunger, there are likely other aspects of hyperphagia that are present. In this blog, we'll discuss some of  the nuance involved in hyperphagia in PWS.

This blog is based on the Research Town Hall presented by the FPWR Research team at the FPWR 2018 conference. You can watch the complete presentation by clicking on the embedded video. In case you don't have time to watch the full video, we've summarized each presentation below.

Our FPWR funded researchers are working hard to eliminate the challenges of PWS. How about spreading some cheer this season by sending a holiday card thanking them for their work? Mailing addresses for FPWR's currently funded researchers are below or can be downloaded here. 

The Foundation for Prader-Willi Research and the Angelman Syndrome Foundation are funding the world’s largest newborn screening study for four rare genetic disorders: Angelman, Prader-Willi, Fragile X and Dup15q syndromes. The Victorian Medical Research Acceleration Fund this year also contributed $100,000 toward the project.

On Wednesday, October 10th, the inaugural Live Life Full DC took place at Army Navy Country Club in Arlington, VA. The event raised nearly $300,000 for PWS research and included guest speakers President Bill Clinton, Special Olympic Chairman, Tim Shriver, and Gold Medalist Special Olympic Swimmer, Meghan Michie.

The event was hosted by Mary & Ian Alberg with the help of their honorary committee members Kristin White, Dominque Deleage, and Rachel Petterson. 

In celebrating five years of success with its comprehensive patient registry, the National Organization for Rare Disorders (NORD) has called special attention to FPWR’s PATH for PWS study.

The study stems from improvements made to the NORD registry that allow greater collaboration and engagement among stakeholders and experts to accelerate the pace of research for rare conditions.

FPWR is happy to announce that Lisa Matesevac has joined our team as our coordinator for the PATH for PWS Study, facilitating families’ involvement in the study. PATH for PWS will help us understand the medical complications that people with PWS experience, and what factors can decrease or increase their risk. 

More than 150 scientists, clinicians, industry representatives and community members attended FPWR’s 2018 Research Symposium, making it one of the largest collaborative Prader-Willi syndrome (PWS) research meetings held to date. The single day program included 26 oral presentations and 22 posters representing all areas of science from discovery to preclinical and clinical research.

“The quality of science shared at this year’s meeting was remarkable. PWS research encompasses a wide range of disciplines and the research presented at the Symposium reached across the spectrum, advancing topics that are important to the PWS community,” said FPWR Director of Research Programs Theresa Strong, Ph.D.

The Foundation for Prader-Willi Research (FPWR) and Zafgen are pleased to announce that enrollment is now open for PATH for PWS, a natural history study intended to better understand serious medical events in Prader-Willi syndrome (PWS) and evaluate how PWS-related behaviors change over time. The data from this study is intended to inform the development and clinical trial design of potential new treatments for PWS. Those interested in participating can find more information about the study and how to enroll at www.PATHforPWS.com.

Several NEW PWS trial opportunities are now available. FPWR maintains the latest clinical trial information so that you can stay abreast of trial opportunities. Some trials require in-person visits while others can be completed remotely. Some trials are testing new drugs while others are intervention (non-drug) or natural history studies, like the PWS Weight Study described below.