Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired disorder that leads to the premature death and impaired production of blood cells.
- Organizations supporting this disease
About AKARI on Alport Syndrome
Paroxysmal Nocturnal Haemoglobinuria (PNH)
PNH is an ultra-rare, life-threatening and debilitating disease of the blood with an estimated 8,000 – 10,000 patients globally. Due to an acquired genetic deficiency, uncontrolled complement activation in PNH patients allows their own complement system to attack and destroy blood cells, leading to life-threatening complications.
Patients with PNH suffer from chronic complement activation and destruction of some of their blood cells, known as hemolysis, caused by the C5 cleavage product C5b-9 (the membrane attack complex). This hemolysis is associated with further clinical symptoms and negative outcomes, including kidney disease, thrombosis (blood clots), liver dysfunction, fatigue, impaired quality of life, recurring pain, shortness of breath, pulmonary hypertension, intermittent episodes of dark-colored urine (hemoglobinuria), and anemia.
About Apellis on PNH
Understanding Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, life-threatening blood disease that is associated with abnormally low (below normal) hemoglobin levels. PNH can appear at any age and in any race or gender, and is most often diagnosed in people in their early 30s.1,2
Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue and difficulty breathing (dyspnea).
In PNH, blood cells lack complement regulatory proteins, so the body recognizes these healthy red blood cells as damaged.1 This leads to uncontrolled activation of the complement cascade, initiated at C3, and results in the destruction of oxygen-carrying red blood cells (hemolysis).3 The complement cascade is a part of the immune system that is responsible for recognizing and eliminating pathogens and damaged cells in the blood.
Alexion on PNH
About Alexion on PNH
- PNH is a chronic, progressive, debilitating, and life-threatening ultra-rare blood disorder characterized by complement-mediated hemolysis (destruction of red blood cells).1,2 PNH can strike men and women of all races, backgrounds, and ages without warning, with an average age of onset in the early 30s