Treating Rare Diseases

Plasma is the straw-colored liquid portion of blood. It contains hundreds of proteins that carry out critical functions in the human body, such as antibodies to fight diseases and clotting factors to regulate bleeding. If a person has insufficient levels of any one plasma protein, his or her body cannot carry out these vital functions, causing a variety of chronic and life-threatening medical conditions. Plasma protein therapies are unique biologic medicines that treat plasma protein deficiencies by replacing a person’s missing or functionally damaged proteins. In the United States, a disease is considered rare if it affects fewer than 200,000 individuals. Plasma protein deficiencies have very small patient populations and can be considered extremely rare.