PATH for PWS Study Highlighted by National Organization for Rare Disorders

In celebrating five years of success with its comprehensive patient registry, the National Organization for Rare Disorders (NORD) has called special attention to FPWR’s PATH for PWS study.

The study stems from improvements made to the NORD registry that allow greater collaboration and engagement among stakeholders and experts to accelerate the pace of research for rare conditions.

A More Powerful Registry

NORD created the Natural Histories Patient Registry Platform, which for five years has tackled the problem of data being diluted across multiple registry platforms. Now, with the addition of the ability to establish sub-studies to capture distinct data related project needs or trends in a specific population, NORD reports it is providing a direct pathway for industry and researchers to partner with NORD and patient organizations.

The expanded functionality unites the power of multiple registries for smaller patient populations (for example, the Global PWS Registry), while ensuring patient communities remain empowered as partners throughout the process.

The registry was built in close collaboration with the FDA, which has allowed the registry to be a user-friendly tool for collecting data urgently needed to better understand rare diseases and patient experiences and ensure new treatment options become available. The registry currently represents 32 registry partnerships, nearly 7,000 active participants, and five years of data collection. 

In a video released by NORD, Janet Woodcock, Director of the Center for Drug Evaluation and Research at the Food and Drug Administration (FDA), discusses the importance of patient registries for rare disease research and urges individuals to participate in NORD’s Registry Program. 

In the video, which can be viewed below, Woodcock explains that there are so many rare disease that it’s difficult for disease prevention advocates to compete among drug manufacturers for investments for new treatments. Through the NORD registry, drug developers can get a clear path to get drugs to market by gaining a clear understanding about the diseases and the people they affect. Woodcock says that this type of data and process is essential for drug development.

Greater Engagement, Starting With FPWR

According to NORD, the first sub-study model on NORD's platform includes a partnership between Zafgen and FPWR for the PATH for PWS study. This natural history study is aimed at better understanding serious medical events in PWS and evaluating how PWS-related behaviors change over time.

This is a four-year study that will specifically evaluate the incidence of serious medical events, prescription medication use associated with medical events of interest, patterns of hyperphagia-related behaviors, as well as additional areas of importance to the PWS community such as changes in body weight, management strategies for hyperphagia and characterization of traits and actions common to individuals with PWS.

Data from this study will inform the development and clinical trial design of potential new treatments. 

Participants are welcome, and enrollment is encouraged. Meet the PATH for PWS coordinator here.

About NORD

The National Organization for Rare Disorders (NORD)® is the leading independent advocacy organization representing all patients and families affected by rare diseases. NORD is committed to the identification, treatment and cure of the 7,000 rare diseases that affect 30 million Americans, or 1 in every 10 people. NORD represents more than 280 disease-specific member organizations and their communities and collaborates with many other organizations in specific causes of importance to the rare disease patient community.

For people with PWS, caregivers and healthcare providers with questions about the PATH for PWS study or enrollment information, visit or contact us via email. 

Enroll in PATH for PWS Today!

Topics: News

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.