Global PNH Patient Registry

The primary aim of the Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry is to conduct a prospectively-planned and efficient natural history study that will result in a more comprehensive understanding of the disease and its course and pace over time. Other registry objectives include the following: - Provide a convenient online platform for participants (or caregivers) to self-report cases of PNH. - Develop a communications registry within the Global PNH Patient registry (e.g., to notify patients of research studies and clinical trials). - Characterize and describe the Global PNH population as a whole, enhancing the understanding of disease prevalence and phenotype as well as the rate of progression of disease characteristics. - Assist the PNH community with the development of recommendations and standards of care. - Be a case-finding resource to be used for researchers who seek to study the pathophysiology of PNH, retrospectively collate intervention outcomes, and design prospective trials of novel treatments.