The new study, the Global PNH Patient Registry *, creates a platform for patients around the world to share information about their PNH experience. By collecting data on as many affected people as possible, the study will serve as an international resource for researchers seeking to improve the lives of patients with PNH.
“The Aplastic Anemia and MDS International Foundation is a 37-year, patient-centered organization providing answers, support and hope to patients with bone marrow failure and their families,” said Janice Frey-Angel, CEO of AAMDSIF. “The Global PNH Patient Registry is an opportunity to give hope to PNH patients around the world by sharing information with each other and with researchers to continue the search for better treatment options and a cure.”
To help raise awareness of PNH and encourage participation in the study, AAMDSIF and its community partners are working with NORD to reach out to the patient community, which in United States alone, about 400 to 500 people are diagnosed with PNH each year. “Our goal is to recruit as many patients, or their parents or legal guardians, as possible,” said Frey-Angel. “The success of the registry depends on broad community participation.”
The Global PNH Patient Registry is a natural history study that consists of electronic surveys to collect information about the patient experience and disease progression. Patients, or their caregivers or guardians, can enter information from anywhere in the world, making it easy for them to participate. The data is anonymized and stored securely in the registry. AAMDSIF may share the data with individuals or institutions conducting research or clinical trials, as approved by the study board which includes scientists, physicians and patient advocates.
“Patient-driven registries are changing the landscape of rare disease research,” said Stephanie Christopher, Associate Director of Research Programs at NORD. “By building strong partnerships within the community and with leading scientific experts, NORD’s registration program is well positioned to fill knowledge gaps and accelerate the development of life-saving discoveries.” IAMRARE® Registry Community! “
PNH is a rare blood disease that affects at least one in a million people. PNH occurs because the surface of a person’s blood cells is missing a protein that protects them from the body’s immune system. This lack of protection causes red blood cells to break down and release hemoglobin. PNH can appear at any age and in any race or sex, but is most commonly diagnosed in people in their 30s and 40s.
For more information, visit PNH.iamrare.org.
* The Global PNH Patient Registry is a collaborative effort between AAMDSIF and NORD, as well as the support of industry partners, Apellis Pharmaceuticals Inc., Genentech, Inc. and BioCryst Pharmaceuticals, Inc.
About Aplastic Anemia and the International MDS Foundation (AAMDSIF)
AAMDSIF is the world’s leading nonprofit healthcare organization dedicated to supporting patients and families living with aplastic anemia, myelodysplastic syndrome (MDS), paroxysmal nocturnal hemoglobinuria (PNH) and related diseases. insufficient bone marrow. The Foundation provides answers, support and hope to thousands of patients and their families around the world.
AAMDSIF is a patient-centered, patient-centered organization serving patients and families throughout the three phases of bone marrow failure disease; their life-changing diagnostic phase, their life-threatening treatment phase, and their life phase with chronic disease throughout their life.
About the National Organization for Rare Diseases (NORD®)
The National Organization for Rare Diseases (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases in the United States. NORD started out as a small group of patient advocates who formed a coalition to unite and mobilize support to pass the Orphan Medicines Act 1983. Since then, the organization has led the way in voicing the needs of the community. diseases, by leading supportive policies, continuing education, advancing medical research and providing patient and family services to those who need them most. With more than 330 disease-specific member organizations, more than 15,000 Rare Action Network advocates in all 50 states, and national and global partners, NORD delivers on its mission to improve the lives of people affected by rare diseases. Visitrarediseases.org.
SOURCE National Organization for Rare Diseases (NORD)