As we wrap up 2025, we’re celebrating a year shaped by real progress… scientific breakthroughs, new treatment pathways, and meaningful improvements in care for people with Prader-Willi syndrome (PWS) and Schaaf-Yang syndrome (SYS).
Every step forward this year was fueled by families, donors, researchers, clinicians, and advocates who push this work ahead every day. Together, we are expanding what’s possible for our loved ones and building a future filled with hope.
Breakthroughs in Treatment Development
A First in PWS: A Treatment for Hyperphagia Approved
For the first time, the FDA approved a treatment for hyperphagia in PWS. DCCR (VYKAT XR) received regulatory approval in March 2025, marking a landmark moment for families who have waited decades for a therapy that targets one of the biggest challenges of PWS.
FPWR is proud to have played a role in this milestone through early investment in the 2014 Phase 2 study and through data from the Global PWS Registry, which showed that VYKAT use was associated with improvements in hyperphagia and behavior.
Momentum in FPWR’s VNS Clinical Trial
Enrollment for FPWR’s first fully sponsored clinical trial is nearly complete. This study is testing whether non-invasive Vagus Nerve Stimulation can help reduce temper outbursts. With more than 80% of the needed participants enrolled, we are getting closer to understanding whether this approach can become another meaningful option for people with PWS.
Improving Care and Daily Quality of Life
Repurposing a Familiar Medication: Guanfacine Shows Benefit
An FPWR-supported clinical trial found that guanfacine, an FDA-approved medication, significantly reduced aggression, skin-picking, and hyperactivity in children and young adults with PWS compared with placebo, with no serious side effects. These results are encouraging and may help guide clinical care immediately.
Guidance on GLP-1 Use in PWS
As interest grows in GLP-1 medications such as Ozempic and Mounjaro, so does the need to understand whether they may be safe and effective for individuals with PWS. This year, FPWR and IPWSO brought experts together to review what we know so far and to outline how best to study how individuals with PWS are responding to GLP-1 medications in the 'real world.'
Research is still limited, but we now have clearer guidance and a roadmap for the next steps in understanding safety and outcomes in PWS.
Protecting Infants: Understanding Swallowing and Aspiration Risk
New research identified risk factors for swallowing challenges in infants with PWS. These findings are an important step toward improving safety, preventing aspiration, and giving families clearer guidance during early care.
Real-World Insights from the Global PWS Registry
More than a decade of registry data continues to shape clinical care. Families and clinicians are using these insights to better understand trends in growth, behavior, medical complications, and how individuals with PWS respond to different treatments.
Tools and Resources for Families
This year, FPWR expanded its library of practical supports, including the new guide Helping Siblings Thrive in a PWS Family, which offers compassionate strategies for nurturing siblings’ well-being.
FPWR-funded research also resulted in more than a dozen peer-reviewed publications, covering topics such as:
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CRISPR-based activation of PWS genes
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Disruption of circadian rhythms in PWS and SYS cells
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Characterization of new PWS mouse models
Each of these studies adds meaningful new knowledge to the scientific community and deepens our understanding of PWS and SYS.
Investing in the Next Breakthrough
$2.1 Million in Research Funding
In 2025, FPWR funded 16 new research projects focused on treatment development, early-stage discovery, and improvements in clinical care. Any one of these projects could be a key step toward the next major breakthrough.
Collecting Patient Data through the PWS-CLIC Database
FPWR’s support of the PWS Clinical Investigation Collaborative (CLIC) has led to the launch of a centralized clinical database where clinicians contribute de-identified patient data. More than 100 patients have already been enrolled. This growing resource is shaping clinical care and supporting future clinical trials.
Advancing Gene Therapy Approaches
FPWR-supported scientists at Duke University made an important discovery: they were able to activate a key epigenetic switch that turns on silenced PWS genes on chromosome 15. This CRISPR-based finding is an early but meaningful step toward developing treatments that may one day address the root cause of PWS.
In parallel, Dr. Marnie Blewitt’s laboratory is exploring whether removing SMCHD1 can reactivate genes in the PWS region. Together with other FPWR-supported gene therapy studies, these advances point toward several possible paths for future PWS gene therapy.
Looking Ahead to 2026
In the coming year, we anticipate:
- The completion of our VNS4PWS clinical trial
- Continued data collection from people using VYKAT XR
- Expansion of the CLIC database
- Progress on multiple FPWR-funded treatment development projects
- Advances in gene activation research
Every breakthrough we celebrated in 2025 happened because of you. Your support fuels research, accelerates treatment development, and brings real hope to families.
Together, we are building a future where people with Prader-Willi syndrome and Schaaf-Yang syndrome have access to meaningful therapies, improved care, and the chance to reach their fullest potential.
From all of us at FPWR, thank you. 💙
