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.

Your blood is:

  • 55%


  • 44%


  • 1%

    white blood cells & platelets

Your plasma is:

  • 92%


  • 7%


  • 1%

    other solutions

Proteins in your plasma:

  • Alpha-1 proteinase inhibitor
  • C1 esterase inhibitor
  • Clotting factors
  • Immunoglobulins (antibodies)

Patients Treated with Plasma Protein Therapies in the U.S.

U.S. Patients treated annually (estimates)

Primary immunodeficiency diseases

Causes & symptoms
  • Caused by missing immunoglobins (antibodies)
  • Antibodies control the immune system and prevent illness
  • Patients are chronically ill from severe, persistent, recurrent infections
U.S. Patients treated annually (estimates)

Bleeding disorders (e.G. Hemophilia)

Causes & symptoms
  • Caused by missing clotting factor protein
  • Clotting factors control bleeding
  • Patients cannot regulate bleeding
  • Can be fatal if bleeding occurs in brain or vital organs
U.S. Patients treated annually (estimates)

includes recombinant therapies
* American Thrombosis & Hemostasis Network

Chronic inflammatory demyelinating polyneuropathy

Causes & symptoms
  • Cause not certain; immune system attacks nerve coating
  • Messages from the brain aren’t delivered to the body if nerve coating is damaged
  • Patients experience progressive weakness, loss of limb function, and disability
U.S. Patients treated annually (estimates)

Alpha-1 antitrypsin deficiency

Causes & symptoms
  • Caused by missing Alpha-1 Proteinase Inhibitor
  • Alpha-1 Proteinase Inhibitor protects the lungs
  • Patients have chronic emphysema and liver damage
U.S. Patients treated annually (estimates)

Hereditary angioedema

Causes & symptoms
  • Caused by missing C1 esterase inhibitor protein (C1-INH)
  • C1-INH helps regulate inflammation
  • Patients have edema (severe swelling)
  • Can be fatal if airway obstructed
U.S. Patients treated annually (estimates)

includes recombinant therapies
* American Thrombosis & Hemostasis Network

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