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 ?
has launched with an initial investment led by the venture philanthropy arm of the Alpha-1 Foundation and a private investor with Alpha-1 Antitrypsin Deficiency (Alpha 1), the company said.
to expand development of induced pluripotent stem cell (iPSC) lines created from tissue donated by patients with Alpha-1 Antitrypsin Deficiency.
Halperin, who is producing a new book on Jackson's life, insists that the Thriller star is suffering from the deadly lung condition Alpha-1 antitrypsin deficiency.
Another two originally assumed to have fatty liver disease were diagnosed with alpha-1 antitrypsin deficiency One child was diagnosed with Wilson's disease, a rare, inherited condition in which a patient cannot fully metabolize copper.
Evaluation of an integrative diagnostic algorithm for the identification of people at risk for alpha-1 antitrypsin deficiency.
Excluded from the review were children, persons with asthma, and those with alpha-1 antitrypsin deficiency.
Key words: alpha-1 antitrypsin deficiency, chronic obstructive pulmonary disease, emphysema, environmental risk factors, gene-environment interactions, genetic testing, liver disease, neutrophil elastase, occupational health.
The funding will allow AGTC to continue development of its Phase 2 program in Alpha-1 Antitrypsin Deficiency (Alpha-1) and initiate full development of potential treatments for two orphan ophthalmology indications, Achromatopsia (ACHM) and X-Linked Rentinoschisis (XLRS).
The global Alpha-1 Antitrypsin Deficiency treatment market will augment at formidable CAGR of 10.
M2 EQUITYBITES-January 20, 2016-Arrowhead Research wins EMA's orphan drug designation for ARC-AAT for treatment of liver disease associated with alpha-1 antitrypsin deficiency
The company will use the funds to continue developing its phase 2 programme in Alpha-1 Antitrypsin Deficiency treatment and to launch full development of potential treatments for orphan ophthalmology indications Achromatopsia (ACHM) and X-Linked Rentinoschisis (XLRS), it explained.

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