Summary
Do GLP-1s work for PWS? Drs. Rubenstein and Stokes are surveying health records from 14,000 individuals living with PWS from 2014 to 2025 to identify which GLP-1s have been prescribed, how well they worked, and if there were any side effects.
Lay Abstract
Over the past decade, new weight-management medications such as Wegovy and Zepbound (a class of drugs called GLP-1s) have become widely used in the United States. These medications are of great interest to the Prader-Willi Syndrome (PWS) community because they might help reduce hyperphagia (excessive hunger). So far, only a few small studies have evaluated GLP-1s in people with PWS, and the results have been mixed. Research has been difficult because PWS is rare, many families do not have the chance to participate in clinical studies, and the medications themselves are new and changing quickly. As a result, families and clinicians do not yet have the information they need to know whether, when, and for whom GLP-1s should be used in PWS. Through a partnership with the Foundation for Prader Willi Research and Komodo Health, investigators at Boston University will analyze a large database of anonymous healthcare records from approximately 14,000 people with PWS living in the United States. Using data from 2014-2025, the team will examine who is being prescribed GLP-1s, how long they are being used, how well they work for weight and health, and what side effects may occur. The project will pay close attention to differences by age, race, ethnicity, geographic region, and economic status to ensure that the experiences of all people with PWS are represented. By providing real-world evidence on GLP-1 use in PWS, this work will offer clearer guidance to families and clinicians and support efforts to help people with PWS live healthier, fuller lives
Funded Year:
2026
Awarded to:
Eric Rubenstein, PhD, ScM and Andrew Stokes, MD
Amount:
$199,800
Institution:
Boston University School of Public Health
