alpha-1 antitrypsin deficiency


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alpha-1 antitrypsin deficiency

An inherited condition–frequency, ±1:10,000, characterized by low or absent production of alpha-1 antitrypsin, an enzyme which is critical to tissue remodeling Clinical The PiZZ phenotype is characterized by early-onset emphysema and liver-related symptoms—jaundice, cholestasis, fatigue, cirrhosis, liver failure, ascites, mental changes, GI bleeding, and ↑ risk of liver CA Treatment IV or nebulized prolastin if COPD, for direct delivery to lungs; alpha-1 proteinase inhibitor; liver transplant; the gene for A1AT may be transferred via adenoviruses to the lung epithelium; following transfer, A1AT mRNA is expressed as functioning A1AT Management Prolastin, O2, antibiotics, phenobarbital or cholestyramine for jaundice and itching, liver transplant
References in periodicals archive ?
Genetic and environmental modulation of COPD in alpha-1 antitrypsin deficiency.
for a new drug, GLASSIA[TM] [Alpha 1-Proteinase Inhibitor (Human)], the first and only liquid, ready-to-use treatment for alpha-1 antitrypsin deficiency (AATD) in the United States.
The Company's most advanced programs in development are treatments for Alpha-1 antitrypsin deficiency, a disease causing a progressive loss of lung function, and Leber's Congenital Amaurosis, an inherited condition causing early blindness.
Plasma protein therapies, which include plasma-derived therapies and recombinant blood clotting factors (a biotechnology product), are used every day to treat people with bleeding disorders, such as hemophilia, that causes painful internal bleeding and debilitating joint damage; primary immunodeficiency diseases, which prevent a person from fighting off even common infections; and alpha-1 antitrypsin deficiency, also known as genetic chronic obstructive pulmonary disease (COPD), a disease that severely damages the liver and lungs.
The annual research grants are part of the Alpha-1 Foundation's ongoing commitment to increase the understanding of alpha-1 antitrypsin deficiency, a genetic disorder in which low levels of the A1PI protein can result in liver damage and emphysema.
a global, diversified healthcare company ("Baxter") announced today that they have entered into an exclusive distribution and manufacturing agreement for Kamada's liquid, ready to use, intravenous Alpha-1 Antitrypsin Product for the treatment of hereditary Alpha-1 Antitrypsin Deficiency approved by the USFDA on July 1st, 2010 for marketing under the trade name "Glassia[TM]".
An estimated 20 million Americans carry the gene which causes Alpha-1 Antitrypsin Deficiency, a disorder that can cause permanent and even fatal organ damage.
Special Note: AlphaNet and the Alpha-1 Foundation work on behalf of those afflicted with Alpha-1 Antitrypsin Deficiency (A1AD).
The program is named in honor of Dr Carl-Bertil Laurell, who first described Alpha-1 Antitrypsin Deficiency (AAT deficiency) in 1963.
MIAMI -- The Alpha-1 Foundation today challenged a newly published review which questions the value of augmentation therapy for Alpha-1 Antitrypsin Deficiency (Alpha-1).
Nationwide, tens of thousands of individuals rely on plasma protein therapies to treat rare, chronic diseases and disorders, which include hemophilia and other bleeding disorders, primary immunodeficiency diseases, alpha-1 antitrypsin deficiency, Kawasaki disease, and certain autoimmune and neurological disorders," said Birkofer.
David Tsur added, "The Biologics License Application for AAT-IV for the treatment of alpha-1 antitrypsin deficiency was recently accepted for review by the US Food and Drug Administration and a decision on its approval is expected in Q2 2010.

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