Established in 2015 by FPWR, in collaboration with international Prader-Willi syndrome (PWS) experts and community stakeholders, the Global PWS Registry is a longitudinal natural-history registry designed to advance research and accelerate therapeutic development. The Registry supports researchers by providing comprehensive real-world data on individuals with PWS, facilitating clinical trial recruitment, and identifying unmet research and treatment needs.
Hosted on the National Organization for Rare Disorders (NORD) Registry Platform, the Global PWS Registry includes more than 5,000 participants from 28 countries. Approximately 75% of participants reside in the United States, representing an estimated 10% of the U.S. PWS population, with additional participation from Canada, the United Kingdom, Australia, and other countries.
Registry Data and Longitudinal Follow-Up
The Registry collects comprehensive caregiver-reported data across the lifespan, including:
- Pregnancy and birth history
- Diagnostic history
- Growth and development
- Medical history and comorbidities
- Behavioral and mental health
- Medications and supplements
- Non-pharmacologic interventions
- Quality of life
Participants are encouraged to update medical information annually, creating a longitudinal dataset that enables researchers to evaluate disease progression, treatment patterns, and changes over time. Additional longitudinal assessments capture behavioral outcomes and other validated measures relevant to clinical research.
Research Applications
The Global PWS Registry supports a wide range of research activities, including natural history studies, observational research, outcome measure development, and clinical trial planning.
Registry data have supported several important initiatives, including:
- PATH for PWS, a four-year longitudinal study of serious medical events in individuals with PWS. This dataset provides a reference to better understand the scope and frequency of these events in the PWS population
- Development and validation of clinical outcome measures
- Caregiver burden assessments and other studies informing therapeutic development
Because Registry data can be linked across multiple domains—including medical history, behavioral assessments, and demographic information—it provides a rich resource for retrospective analyses and hypothesis generation.
Data Access and Governance
De-identified, aggregated data from the Registry is being shared with academic and industry collaborators conducting retrospective studies or conducting research and/or clinical trials on new therapies. Third parties are granted access to de-identified Registry data according to the guidelines of the Global PWS Registry Advisory Board, and an independent IRB provides ethical oversight for Registry activities. Third parties seeking to recruit participants for research studies through the Registry must demonstrate evidence of IRB approval of the research study that will be recruited.
For More Information
For more information, email Theresa Strong, Ph.D., Director of Research Programs, or Jessica Bohonowych, Ph.D., Associate Director of Research Programs.
Caregivers in the PWS community can learn more about the Registry and participation on FPWR's Global PWS Registry page.