Patients suffering from PNH benefit from Paradoxical Nocturnal Hemoglobinuria Therapeutics, which decreases their risk of blood clots and increases their quality of life.

PNH (paroxysmal nocturnal hemoglobinuria) is a rare disorder in which red blood cells break apart prematurely. It is an acquired hematopoietic stem cell disorder. Hematopoietic stem cells are produced in the bone marrow, the spongy centre of the body's long bones. These cells develop into red blood cells, white blood cells, and platelets. Some hematopoietic stem cells in PNH patients are defective, resulting in defective blood cells. These Paroxysmal Nocturnal Hemoglobinuria Therapeutics red blood cells are extremely vulnerable to premature destruction by the complement system, a component of a person's own immune system.

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