Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder in which red blood cells break apart prematurely.
The specific symptoms and progression of the disorder vary greatly from one patient to another. Some individuals may have mild symptoms that remain stable for many years; others may have serious symptoms that can progress to cause life-threatening complications.
A diagnosis of PNH may be suspected in individuals who have symptoms of intravascular hemolysis (e.g., hemoglobinuria, abnormally high serum LDH concentration) with no known cause
The main diagnostic test for individuals with suspected PNH is flow cytometry, which is a blood test that can identify PNH cells.
Empaveli (pegcetacoplan)
Empaveli (pegcetacoplan)
Empaveli (pegcetacoplan) is a complement inhibitor indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
Patient resources
National Organization for Rare Disorders (NORD) supports patient advocacy, education, and research.
The Aplastic Anemia and MDS International Foundation, Inc provides education, resources, and support services for patients with PNH.
Genetic and Rare Diseases Information Center (GARD) is a public health resource that aims to support people living with a rare disease and their families.
