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  2. Alpha-1 antitrypsin deficiency - Wikipedia

    en.wikipedia.org/wiki/Alpha-1_antitrypsin_deficiency

    Alpha-1 antitrypsin deficiency (A1AD or AATD) is a genetic disorder that may result in lung disease or liver disease. [1] Onset of lung problems is typically between 20 and 50 years of age. [ 1 ] This may result in shortness of breath , wheezing , or an increased risk of lung infections .

  3. Vanishing bile duct syndrome - Wikipedia

    en.wikipedia.org/wiki/Vanishing_Bile_Duct_Syndrome

    [1] Signs and symptoms. The presentation is dependent upon the underlying cause. The course can be rapid or chronic. ... Alpha 1 antitrypsin deficiency;

  4. Alpha-1 antitrypsin - Wikipedia

    en.wikipedia.org/wiki/Alpha-1_antitrypsin

    Alpha-1 antitrypsin or α 1-antitrypsin (A1AT, α 1 AT, A1A, or AAT) is a protein belonging to the serpin superfamily. It is encoded in humans by the SERPINA1 gene.A protease inhibitor, it is also known as alpha 1 –proteinase inhibitor (A1PI) or alpha 1-antiproteinase (A1AP) because it inhibits various proteases (not just trypsin). [5]

  5. PAS diastase stain - Wikipedia

    en.wikipedia.org/wiki/PAS_diastase_stain

    PAS diastase stain is also used to identify alpha-1 antitrypsin globules in hepatocytes, which is a characteristic finding of alpha-1 antitrypsin deficiency. [2] PAS diastase stain is also used in diagnosing Whipple’s disease , as the foamy macrophages that infiltrate the lamina propria of the small intestine in this disease possess PAS ...

  6. Alnylam Presents New Pre-clinical Data on RNAi Therapeutics ...

    www.aol.com/news/2012-11-14-alnylam-presents-new...

    Alpha-1 antitrypsin deficiency is an autosomal disorder that results in disease of the lungs and liver, and afflicts roughly 10,000 patients worldwide. AAT is a liver-produced serine proteinase ...

  7. Vertex Pharmaceuticals Inc (NASDAQ: VRTX) has advanced its investigational program targeting alpha-1 antitrypsin deficiency (AATD), a rare genetic disease characterized by a protein folding defect ...

  8. Elastase - Wikipedia

    en.wikipedia.org/wiki/Elastase

    Elastase is inhibited by the acute-phase protein α 1-antitrypsin (A1AT), which binds almost irreversibly to the active site of elastase and trypsin. A1AT is normally secreted by the liver cells into the serum. Alpha-1 antitrypsin deficiency (A1AD) leads to uninhibited destruction of elastic fibre by elastase; the main result is emphysema.

  9. Neonatal cholestasis - Wikipedia

    en.wikipedia.org/wiki/Neonatal_cholestasis

    Additional symptoms may vary based on the cause of neonatal cholestasis. For example, if a patient’s symptoms are caused by a choledochal cyst, they may present with abdominal pain, vomiting, and a palpable abdominal mass. [4] Patients with alpha-1 antitrypsin deficiency may present with hepatomegaly and elevated liver enzymes. [9]