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4 Reasons to be Excited for PWS Research in 2021

Our Executive Director shares her sources of hope for 2021, thanks to meaningful advances in research as we work towards treatments and a cure for PWS!

IMG_2602-JPG-1My son with PWS turns 12 years old this year. He experiences many of the challenges of PWS and, like every parent, I dream of the day when he can live a FULL and independent life. This is why I am as excited as ever as we enter 2021 … 

  1. Two PWS clinical trials for hyperphagia were completed in 2020 with promising results.

    If approved by the FDA these will become the first new treatments for PWS since growth hormone approved more than 20 years ago! Additionally, a pivotal phase II trial of Pitolisant has been launched to address wakefulness inn PWS. FPWR will continue to work closely with each of study sponsors to advance PWS trials in 2021.

  2. Vagus Nerve Stimulation (VNS) has been shown to improve behaviors in individuals with PWS.

    The device has been studied in a small number of PWS individuals both as an implanted device and an external, wearable device, and in both instances, behaviors were markedly improved. Your donations have enabled this work and in 2021 FPWR will advance a clinical trial of VNS in the United States with the aim of collecting the safety and efficacy data needed to make this technology available to our loved ones with PWS.

  3. This past year alone, FPWR-funded researchers have identified 3 NEW potential therapeutic targets, suggesting new possibilities for developing novel treatments for PWS:

    • Dr. Potts and Reiter have described a broken cellular process in PWS. This finding sets the stage for screens to identify drugs that may address many of the symptoms of both PWS and SYS in a single approach.
    • Dr. Carmichael has used stem cells to identify a set of genes that are processed and expressed differently in PWS compared to typical neurons, establishing new targets for therapeutic development.
    • Dr. Schaaf identified a pathway that is overactive in SYS and may be amenable to modification with drug therapy.
  4. In addition to bringing together experts from across many fields of research, FPWR is working closely with more than a dozen companies interested in developing therapeutics for PWS.

    These collaborations are allowing us to accelerate our work as we drive forward towards treatments for PWS.

Despite the challenges of 2020, with your support, we have made meaningful advances as we work towards treatments and a cure for PWS. I know with great certainty that we are getting closer to answers. There will be treatments for our loved ones with PWS. They are coming. And we will keep eliminating the challenges until they have all been overcome.

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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.