As 2024 draws to a close, we at the Foundation for Prader-Willi Research (FPWR) are reflecting on a year filled with progress, promise, and hope. This year’s achievements wouldn’t have been possible without the incredible dedication of our community—researchers, families, donors, and advocates—who share our vision for a brighter future.
With your invaluable support, we continue to push forward, step by step, toward a brighter future for those affected by PWS and SYS.
Fueling Innovation
This year, your support helped FPWR fund over two dozen cutting-edge research grants, pushing the boundaries of PWS and SYS science. These grants represent more than just funding—they reflect a commitment to innovation, collaboration, and discovery.
Through these projects, researchers are exploring critical areas, from the genetic underpinnings of PWS and SYS to novel therapeutic strategies. Each grant is a step closer to our ultimate goal: better treatments and, one day, a cure.
Pioneering Breakthroughs
Thanks to your belief in what’s possible, FPWR has continued to champion research that leads to transformative breakthroughs. This year brought exciting progress in key areas:
- Gene Therapy: FPWR-funded researchers are making strides in gene therapy, working to address the genetic disruptions at the root of PWS and SYS. These efforts could lay the foundation for therapies that go beyond symptoms to tackle the underlying causes.
- New Screening Technology: A newly developed cellular assay now allows us to screen thousands of compounds, identifying those that can restore function in PWS cells. Promising compounds from this process will advance into FPWR’s drug pipeline, offering hope for innovative treatment options.
Advancing Treatments
With your support, FPWR continues to back essential clinical trials, a cornerstone of our mission, bringing promising treatments for PWS closer to reality.
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DCCR Progress: In 2014, FPWR made a critical early investment in the development of DCCR, a potential treatment for hyperphagia, one of the most challenging symptoms of PWS. Today, we are on the brink of a historic milestone—DCCR’s new drug application has been submitted to the FDA, and we eagerly await their decision by the end of the year.
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Expanding Treatment Options: Beyond DCCR, several promising drugs and devices are in active testing for their ability to address challenges like temper outbursts, daytime sleepiness, anxiety, and hyperphagia. Each of these trials represents another step toward a comprehensive toolbox of therapies designed to help individuals with PWS live fuller, more independent lives.
Through these efforts, we’re not just working to treat symptoms—we’re building hope for a future filled with possibilities.
Improving Quality of Life
FPWR’s mission extends beyond treatment development to improving quality of life for individuals with PWS and their families. This year, we saw significant progress in enhancing care and fostering meaningful connections.
- Better Infant Care: Data from the Global PWS Registry revealed that nasogastric (NG) tubes may be a safer option for newborns with PWS compared to gastrostomy (G) tubes, which carry higher complication rates. This finding is already helping shape medical care for the youngest members of our community.
- Empowering Social Growth: Through a partnership with PWSA-USA, access to the BOSS social skills curriculum has expanded, providing teens and adults with PWS opportunities to build essential skills and form lasting connections.
By addressing the diverse needs of the PWS community, we’re helping individuals at every stage of life achieve their goals and unlock their potential.
Looking Forward to 2025
As we reflect on this year’s progress, we’re equally excited about what lies ahead. The future is filled with promise, and 2025 may be one of our most impactful years yet. Here’s what we’re looking forward to:
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Potential Approval of DCCR: The FDA’s decision could make DCCR the first approved treatment for hyperphagia in PWS.
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New Clinical Trials: In 2025, individuals with PWS will have the opportunity to participate in a clinical trial for ARD-101, a potential new therapy targeting hyperphagia.
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Trial Results on the Horizon: Results from ongoing trials for VNS (FPWR), Carbetocin (Acadia), and Pitolisant (Harmony) are expected to provide new insights into treatment options.
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Advancing Genetic Therapy: As genetic therapies for related disorders like Angelman and Rett syndromes move through clinical trials, they lay the groundwork for what could one day be possible for PWS and SYS.
With these exciting developments on the horizon, the future has never looked brighter for our community.
Thank You for Making Progress Possible!
None of these achievements would be possible without your support. Together, we’re building a future where individuals with PWS and SYS have access to effective treatments, enhanced quality of life, and boundless opportunities. As we move to 2025, we’re grateful to have you by our side, fueling hope and driving progress for the PWS & SYS community.
