[PRESS RELEASE] FPWR and NORD launch the Global Prader-Willi Syndrome Registry

WASHINGTON and PASADENA, Calif., May 4, 2015 /PRNewswire-USNewswire/ -- The Foundation for Prader-Willi Research (FPWR) and National Organization for Rare Disorders (NORD)® today launched the Global Prader-Willi Syndrome Registry, a new database to accelerate research and cures for the rare disease Prader-Willi Syndrome.

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.

NORD President and CEO Peter L. Saltonstall said the Global PWS 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 Foundation for Prader-Willi Research Executive Director Susan Hedstrom.  "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 Global PWS 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 is currently 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 NORD Director of Information Technology Vincent Tsugranes.

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."[1]

"The Global PWS Registry is the next step in Prader-Willi research and it will advance PWS research faster than ever before," concluded Hedstrom.

For more information, visit www.pwsregistry.org.

About Foundation for Prader-Willi Research (FPWR) The Foundation for Prader-Willi Research was established in 2003 by a small group of parents who saw the need to foster research that would help their children with Prader-Willi syndrome lead more healthy and fulfilling lives. Today, FPWR is composed of hundreds of parents, family members, researchers, and others who are interested in addressing the many issues related to PWS, including childhood obesity, developmental delay, psychiatric disorders, and autism spectrum disorders.

The mission of FPWR is to eliminate the challenges of Prader-Willi syndrome through the advancement of research. High-quality research will lead to more effective treatments and an eventual cure for this disorder. By working together, we intend to free our loved ones from the burdens of PWS, allowing them to lead full and independent lives.

About National Organization for Rare Disorders (NORD)® Established in 1983, the National Organization for Rare Disorders (NORD)® is the primary nonprofit organization representing all patients and families affected by rare diseases in the U.S.  NORD is committed to the identification, treatment and cure of all 7,000 rare diseases that affect 30 million Americans, or 1 in every 10 people.  NORD provides programs of advocacy, education, research and patient/family services to improve the lives of all people living with rare diseases. NORD represents more than 220 disease-specific member organizations and partners with many other organizations in specific causes of importance to the rare disease patient community. Join NORD at www.rarediseases.org/ and on Twitter at @RareDiseases.

[1] Woodcock, J. "The more we know about rare diseases, the more likely we are to find safe and effective treatments." FDAVoice (Oct. 23, 2014)

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  SOURCE National Organization for Rare Disorders (NORD)  

Topics: Research

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 natural history of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she hosted her first One SMALL Step walk in 2010 and began the development of the One SMALL Step walk program which now raises over $1.5 million a year for PWS research.