Alpha 1 is the common name for a genetic condition called Alpha 1 Antitrypsin Deficiency. If you have Alpha 1, your body is unable to produce a protein known as Alpha 1 Antitrypsin, or AAT for short. This protein plays an important role in protecting your lungs from damage due to excessive inflammation. Without this protein present in your blood, your lungs may weaken over time and place you at a higher risk for developing serious diseases, such as early-onset emphysema often misdiagnosed as chronic obstructive pulmonary disease (COPD). An abnormal amount of AAT in the liver can also cause liver disease.
Patients & Public Health
Alpha 1 Antitrypsin Deficiency
Hereditary Angioedema (HAE) is a rare hereditary disease that can cause attacks of swelling, and often pain, in specific parts of the body including the stomach, hands, feet, arms, legs, genitals, throat, and face. Depending on the severity of the disease, some people will have many attacks each month, while others will go months without an attack.
People with HAE are missing or have low levels of a protein called C1 esterase inhibitor (C1-INH); in some cases, the C1-INH levels are sufficient but the protein does not function properly. The defect with C1-INH lies within a person's genetic code, which is why HAE runs in families.
Types of HAE
Hereditary Angioedema is classified into three types based on what problem the genetic defect causes.
Type I - Low levels of C1-INH in the body; this is the most common form of HAE.
Type II - Normal levels of C1-INH, but it does not function normally; this accounts for ~15% of people with HAE.
Type III - Normal levels of C1-INH and it functions normally; this is extremely rare and not well understood.
Hereditary Bleeding Disorders
Bleeding disorders are a group of conditions that result when the blood cannot clot properly. As a result, people with bleeding disorders experience extended bleeding after injury, menstruation, trauma, or surgery, and in many situations depending on the severity, spontaneous bleeding into joints, muscles, or other parts of their bodies.
Improper clotting results from defects in blood components, such as clotting proteins and/or platelets. The body produces 13 different clotting proteins, also called clotting factors. If any of these clotting factors are missing or not working properly, a bleeding disorder can result. Bleeding disorders are quite rare, and some bleeding disorders such as hemophilia, can be congenital (hereditary) or acquired. Congenital bleeding disorders are caused by defects or damage in the genes and are present at birth. They can be inherited or appear due to a genetic mutation. Acquired bleeding disorders are those that develop after birth or spontaneously occur in the individual.
Treatment varies, depending on the condition and its severity. For some bleeding disorders, there are treatments that can be taken at home to prevent or treat bleeds. With appropriate education and treatment, people with bleeding disorders can live full and productive lives.
Immunodeficiency & Autoimmune Diseases
Your immune system protects your body against dangerous organisms, such as viruses, bacteria and cancer cells. When these organisms infect an otherwise healthy body, the immune system responds by producing antibodies and special blood cells (called lymphocytes) that attempt to eradicate the infection. When your immune system fails to respond adequately to infection, it's called an immunodeficiency, and you may be immunocompromised. People may also suffer from the opposite condition, an overactive immune system that attacks healthy cells as though they were foreign bodies, and that is called an autoimmune response. Both scenarios can create potentially life-threatening conditions.
Immunoglobulin, also known as Ig, is a component of healthy human blood plasma. At CSL Behring, we’ve developed several Immunoglobulin (Ig) replacement therapies to treat rare conditions related to the immune system in which Ig is not present in sufficient quantities. In addition, our product portfolio includes hyperimmune immunoglobulins which are especially high in antibodies against certain diseases, such as tetanus, varicella, rabies, and hepatitis A and B.
Iron Deficiency & Iron Deficiency Anaemia
Iron deficiency and iron deficiency anaemia are highly common conditions present in up to one third of people worldwide.1
Iron is a fundamental mineral needed to produce haemoglobin, a protein in red blood cells that carries oxygen around the body. Iron deficiency indicates a condition in which iron levels are below the minimal threshold.
If iron levels fall too low and are not treated, the body is unable to produce an adequate amount of haemoglobin and healthy red blood cells. This condition is called iron deficiency anaemia which results in symptoms affecting quality of life.
Educational materials about iron deficiency are available for the general public at TakeIronSeriously.com
Non-dialysis chronic kidney disease (CKD) is relatively common among adults, with prevalence of up to 13% and rising as the population ages. People living with CKD face a long and difficult journey associated with multiple and varying conditions and complications that severely impact quality of life and life expectancy. Although CKD cannot be completely reversed, medication is often used to treat associated complications and slowdown further kidney damage.
CSL Seqirus is a transcontinental partner in pandemic preparedness and a major contributor to the prevention of influenza globally. We utilize both egg and cell-based technologies for influenza vaccine production and have developed the only adjuvanted seasonal influenza vaccine for elderly populations.