Global PNH Patient Registry

The Aplastic Anemia and MDS International Foundation (AAMDSIF) and the National Organization for Rare Disorders (NORD®) have launched the largest-ever study to research Paroxysmal Nocturnal Hemoglobinuria (PNH).

The new study, the Global PNH Patient Registry*, creates a platform for patients around the world to share information about their experience with PNH. In gathering data from as many affected individuals as possible, the study will serve as an international resource for researchers looking to improve the lives of PNH patients.

To help drive awareness of PNH and encourage participation in the study, AAMDSIF and its community partners are working in collaboration with NORD to reach the patient community, which in the United States alone consists of around 400 to 500 individuals diagnosed with PNH each year. “Our goal is to enroll as many patients, or their parents or legal guardians, as possible,” said Janice Frey-Angel, CEO of AAMDSIF. “The success of the registry is dependent upon 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 to participate. The data is made anonymous 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’s governing board that includes scientists, doctors and patient advocates.

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*The Global PNH Patient Registry is a collaborative effort between AAMDSIF and NORD, along with the support of industry partners, Apellis Pharmaceuticals, Inc., Genentech, Inc., and BioCryst Pharmaceuticals, Inc.