The National Organization for Rare Disorders (NORD) is celebrating five years of its IAMRARE Registry Program’s success, as well as the creation of new collaborations and models of engagement, including one for Prader-Willi syndrome (PWS) research.
NORD is the leading nonprofit organization that represents the 30 million Americans with rare diseases. The organization has developed a patient registry platform that allows researchers from industry, academia, and medicine to establish sub-studies to assess distinct data specifically related to their project needs, or to study specific trends in a subset of the patient population.
This system accelerates the pace of research and promotes the collaboration between researchers, patient organizations, and industry. It was designed to better understand rare diseases and the experiences of patients.
The first sub-study model on NORD’s registry platform includes a partnership between Zafgen and the Foundation for Prader-Willi Research. The PATH for PWS study aims to evaluate the incidence of serious medical events in participants with PWS, while analyzing existing medical information to better characterize the natural history, or disease course, of PWS.
Zafgen is a biopharmaceutical company developing therapies for metabolic disorders, including PWS. The company’s investigational therapy ZGN-1258 is designed to change the way the body uses fat molecules, reducing fat mass and controlling the excessive urge to eat associated with PWS. The therapy is moving closer to clinical trials in humans.
“NORD is proud to provide this vital and enduring resource for the rare disease community,” Peter L. Saltonstall, president and CEO of NORD, said in a press release.
“With 95% of rare diseases still without an FDA-approved treatment or cure, we remain committed to eliminating hurdles that prevent research into rare diseases. We believe our platform and collaborative registry model has the power to do just that,” Saltonstall said.
NORD has so far fostered 32 registry partnerships, with almost 7,000 active participants, and has five years of robust data collection in a number of rare diseases.
Janet Woodcock, director of the Center for Drug Evaluation and Research at the U.S. Food and Drug Administration, echoed the need for models of engagement and support for NORD’s registry program in a video released by NORD.
Individuals are encouraged to participate in NORD’s Registry Program and make an impact on rare disease research.