antitrypsin deficiency

an·ti·tryp·sin de·fi·cien·cy

deficiency of α1-antitrypsin, a serum protease inhibitor, is associated with emphysema and/or liver cirrhosis. By isoelectric focusing, numerous variants have been identified, with different levels of normal activity; autosomal recessive inheritance, caused by mutation in the P1 gene on chromosomal 14q.
Farlex Partner Medical Dictionary © Farlex 2012
References in periodicals archive ?
Further, the analyst believes investor attention should now shift to the company's emerging pipeline, where he sees a "significant opportunity" in the Phase 2-ready alpha-1 antitrypsin deficiency disorder program.
"Additionally, we have rapidly grown our pipeline beyond CF, advancing seven new potential medicines across five disease areas, including beta thalassemia, sickle cell disease, alpha-1 antitrypsin deficiency, APOL1-mediated kidney diseases and pain.
(NASDAQ: ARWR) today announced that it has dosed the first patient in SEQUOIA (AROAAT2001), a potentially pivotal Phase 2/3 clinical study of ARO-AAT, the company's second generation subcutaneously administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD).
Its current pipeline focuses on new and effective treatments for Alpha-1 Antitrypsin Deficiency (Alpha-1, or AATD) and genetic Amyotrophic Lateral Sclerosis (ALS.)
Beyond CF, Vertex has a pipeline of investigational medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney disease.
INBRX-101 is a modified recombinant version of human alpha-1 antitrypsin, or AAT, for the treatment of patients with alpha-1 antitrypsin deficiency, or AATD.
Summary: Looking at the current market trends as well as the promising demand status of the "Alpha 1 Antitrypsin Deficiency Treatment Market", it can be projected that the future years will bring out positive outcomes.
[USPRwire, Fri May 17 2019] Looking at the current market trends as well as the promising demand status of the " Alpha 1 Antitrypsin Deficiency Treatment Market ", it can be projected that the future years will bring out positive outcomes.
Alpha-1 or Alpha-1 Antitrypsin Deficiency is a lack of the protein alpha-1 antitrypsin (AAT) which is produced in the liver.
for POL6014, a clinical stage selective inhibitor of human neutrophil elastase with the potential to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases such as non-cystic fibrosis bronchiectasis (NCFB), alpha-1 antitrypsin deficiency (AATD) and primary ciliary dyskinesia (PCD).
The tot, from Longbenton, North Tyneside, suffers from a rare genetic condition called alpha 1 antitrypsin deficiency (AATD).