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Paroxysmal Nocturnal Hemoglobinuria (PNH) affects about 1.3 per 100,000 people in the U.S., equally in males and females, with median diagnosis age of 30-40 years. The paroxysmal nocturnal hemoglobinuria epidemiology forecast by Expert Market Research indicates that the condition has an equal prevalence in both males and females.
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Expert Market Research's “Paroxysmal Nocturnal Hemoglobinuria (PNH) Epidemiology Forecast Report 2026-2035” offers comprehensive information on the prevalence and demographics of paroxysmal nocturnal hemoglobinuria (PNH). It projects the future incidence and prevalence rates of paroxysmal nocturnal hemoglobinuria (PNH) cases across various populations. The study covers age, gender, and type as major determinants of the paroxysmal nocturnal hemoglobinuria (PNH) population. The report highlights patterns in the prevalence of paroxysmal nocturnal hemoglobinuria (PNH) over time and projects future trends based on multiple variables.
The report provides a comprehensive overview of the disease, as well as historical and projected data on paroxysmal nocturnal hemoglobinuria (PNH) epidemiology in the 8 major markets.
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Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired hematopoietic stem cell disorder caused by somatic mutations in the PIGA gene, resulting in red blood cells deficient in complement-regulating surface proteins (CD55, CD59). The loss of these proteins leads to complement-mediated intravascular hemolysis, bone marrow failure, and thrombosis. PNH affects all ages but most commonly presents in adults aged 30–40. Symptoms include fatigue, anemia, hemoglobinuria, and venous thrombosis. Diagnosis is confirmed via flow cytometry detecting GPI-anchor protein deficiency.
The paroxysmal nocturnal hemoglobinuria (PNH) epidemiology division offers information on the patient pool from history to the present, as well as the projected trend for each of the 8 major markets. Expert Market Research provides both current and predicted trends for the paroxysmal nocturnal hemoglobinuria (PNH) epidemiology scenario by examining a wide range of studies. Additionally, the report covers the diagnosed patient pool for paroxysmal nocturnal hemoglobinuria (PNH) and their trends. The paroxysmal nocturnal hemoglobinuria (PNH) detailed epidemiology segmentation is broken down into specific categories, such as total prevalent cases in males and females, and total diagnosed cases across different age groups and patient pools.

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The paroxysmal nocturnal hemoglobinuria (PNH) epidemiology data and findings for the United States, Germany, Spain, Italy, France, the United Kingdom, Japan, and India are also provided in the epidemiology section.
In the United States, paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare acquired clonal disorder with a prevalence of approximately 1.3 per 100,000 people, corresponding to about 6,000 diagnosed cases. PNH affects both males and females equally, with a median age at diagnosis between 30 and 40 years. Early recognition and diagnosis remain crucial for initiating targeted therapies, including complement inhibitors, to reduce hemolysis, thrombotic complications, and improve quality of life for affected patients nationwide.
Therapeutic advances have transformed paroxysmal nocturnal hemoglobinuria management. Complement inhibitors such as eculizumab and ravulizumab block C5-mediated hemolysis, significantly reducing transfusion dependence and thrombosis risk. Novel proximal inhibitors like pegcetacoplan target C3 for broader complement control. Supportive therapies include anticoagulation, iron, and folate supplementation. Allogeneic bone marrow transplantation remains the only curative option but carries substantial risk. Lifelong complement blockade and genetic monitoring improve survival, making PNH one of the most successfully targeted hematologic disorders in modern medicine.
*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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Base Year |
2025 |
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Historical Period |
2019-2025 |
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Forecast Period |
2026-2035 |
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Epidemiology Statistics Provided |
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