The Foundation for Prader-Willi Research (FPWR), a nonprofit charitable organization, and National Organization for Rare Disorders (NORD), a nonprofit organization representing all patients and families affected by rare diseases in the U.S., have launched the Global Prader-Willi Syndrome Registry, a new database to accelerate research and cures for the rare disease Prader-Willi Syndrome (PWS).
The Global PWS Registry creates a platform for patients around the world to share information about PWS with researchers on developmental history, medical complications and quality of life.
Peter L. Saltonstall, NORD president and CEO, said the registry addresses what is widely recognized as one of the greatest needs and inherent challenges of the rare disease community—natural history data to help medical researchers better understand how diseases develop and progress over time.
"The Global PWS Registry will provide a complete picture of each patient's experience with Prader-Willi," said Susan Hedstrom, FPWR executive director. "We are launching this initiative to help fill the missing link researchers and medical experts need to advance research and get to a cure."
To help drive awareness and participation, FPWR has created a custom Facebook Profile Pic creator that will allow participants to promote the registry: http://www.fpwr.org/pic-creator. This is one of many activities planned for Prader-Willi Awareness Month.
"Our goal is to enroll as many patients, or their parents or legal guardians, as possible," said Hedstrom. "The success of the registry is dependent upon community participation."
The registry consists of annual electronic surveys to collect information about the patient experience. The information is made anonymous and may be shared with individuals or institutions conducting research or clinical trials. Research studies are reviewed and approved by registry's governing board that includes scientists, doctors and parent advocates.
"Another benefit of the Global PWS Registry is its ability to help match participants with potential clinical trials. If your profile matches the needs of an upcoming clinical trial, you will be notified of the opportunity," added Hedstrom.
PWS is a rare genetic disorder that occurs in approximately one out of every 15,000 births. PWS is recognized as the most common genetic cause of life-threatening childhood obesity. It typically causes low muscle tone, short stature, incomplete sexual development, slow metabolism, a constant and chronic feeling of hunger and cognitive challenges. There currently is no cure.
NORD built its registry program with input from patients and patient organizations, researchers and regulators as part of its mission to identify and treat all 7,000 rare diseases.
"We are committed to helping ensure that every rare disease has adequate natural history information and that all rare disease patient organizations have access to the tools they need to advance patient-centered research," said Vincent Tsugranes, NORD director of information technology.
Janet Woodcock, M.D., director of the FDA's Center for Drug Evaluation and Research, has praised NORD's registry initiative as a helpful tool "that protects the security and privacy of personal information, while making valuable information available to a researcher or drug developer interested in creating a new therapy for a rare disease."