Global PWS Registry Debuts as Part of Prader-Willi Syndrome Awareness Month
Washington, D.C. and Pasadena, Calif.—May 4, 2015—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.” read more >
The following statement was issued today by Peter L. Saltonstall, President and CEO of NORD, following the release of the second discussion draft by the House Energy and Commerce Committee for its 21st Century Cures initiative:
The second discussion draft of the 21st Century Cures initiative released today includes a number of encouraging ideas on how to advance the development of new medical therapies. The Committee and its staff deserve praise for efforts to bring these ideas together and for their months of work to make this a collaborative and transparent process.
We are encouraged by many of the ideas included in the second discussion draft. Since the initiative was launched one year ago, NORD has advocated for several provisions included therein, including incorporating the patient perspective through the patient-focused drug development initiative, developing registries of natural history data of rare disease patients, increasing transparency in the expanded access process, and increased funding for NIH, among others.
Nothing is more important to the rare disease community than providing an environment conducive to the development of new therapies and access to them. We will conduct a thorough evaluation and will continue to work closely with the Committee and members of the House and Senate as legislation moves forward.
Peter L. Saltonstall
President and CEO, National Organization for Rare Disorders (NORD)
The cost of orphan drugs, and whether it is approaching a level where sustainability may be an issue, is a topic of intense interest in the rare disease community. However, an important new study reported at the recent American Society of Hematology meeting suggests that the overall budget impact of orphan drugs may be smaller than many people would assume. read more >
If you have supported NORD with a donation at any time during its 31+ year history, we thank you. The wonderful work that NORD has accomplished since 1983 on behalf of rare disease patients and their families wouldn’t have been possible without the support of many caring individuals.
NORD works very hard to be a careful steward of donated funds, keeping operating costs low so that 95 cents of every dollar goes directly to programs and services for patients.
We know that you receive many solicitations for contributions at this time of year, but we believe that NORD has earned a place at the top of your list through its history of dedicated leadership and service.
And we are excited to tell you about two unique opportunities to drive progress for patients in 2015: promoting state-based advocacy through NORD’s Rare Action Network™ (RAN) and advancing research with an innovative new platform NORD has developed for patient registries and natural history studies. read more >
Biologic therapies have been lifesaving and life changing for the patients we represent – more than 30 million Americans with more than 7,000 rare diseases. Literally, hundreds of thousands of rare disease patients have found their lives bettered by biologics and we are collectively grateful to the innovators and companies that have made this possible. read more >
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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