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Diseases

Genetic and Rare Diseases Information Center (GARD)

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Alpha 1-antitrypsin deficiency


Other Names for this Disease

  • A1AT deficiency
  • AAT deficiency
  • AATD
  • Alpha 1 antitrypsin deficiency
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.

Treatment

Management Guidelines

  • GeneReviews provides current, expert-authored, peer-reviewed, full-text articles describing the application of genetic testing to the diagnosis, management, and genetic counseling of patients with specific inherited conditions. Click on the link to view the article on this topic.
  • Orphanet Emergency Guidelines is an article which is expert-authored and peer-reviewed that is intended to guide health care professionals in emergency situations involving this condition.  

Clinical Trials & Research for this Disease

  • The Childhood Liver Disease Research and Education Network (ChiLDREN) is a team of doctors, nurses, research coordinators, and research labs throughout the U.S., working together to improve the lives of people with rare liver diseases through research. Current studies are enrolling people with Alagille syndrome, alpha one-antitrypsin deficiency, bile acid synthesis defects, biliary atresia, cystic fibrosis liver disease, idiopathic neonatal hepatitis, mitochondrial hepatopathies, and progressive familial intrahepatic cholestasis. Click on the link above to learn more.

    Visit the following link to find the participating research center nearest you.
    http://childrennetwork.org/centers.html

  • ClinicalTrials.gov lists trials that are studying or have studied Alpha 1-antitrypsin deficiency. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.

Medical Products

The medication(s) listed in the table(s) below have been approved by the Food and Drug Administration (FDA) for treatment of this condition. The FDA Office of Orphan Products Development designates "orphan products" for those that treat rare diseases affecting fewer than 200,000 Americans. The table(s) below may not be an exhaustive list of drugs or products used to treat this condition. There may be other products available that are not considered orphan products. To search for all FDA approved drugs, visit Drugs@FDA. You can find orphan products used to treat other conditions by searching the Orphan Drug Product Designation database.


Generic Name Alpha1-Proteinase Inhibitor (Human)
Trade Name
(Manufacturer Name)
Prolastin®
(Talecris Biotherapeutics)
Indication
The FDA has approved this product to be used in this manner.
For chronic replacement therapy of individuals having congenital deficiency of alpha1- proteinase inhibitor with clinically demonstrable panacinar emphysema.
More Information about this product Drug Information Portal

Other Names for this Disease
  • A1AT deficiency
  • AAT deficiency
  • AATD
  • Alpha 1 antitrypsin deficiency
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.