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1 dividuals with emphysema secondary to severe alpha1 antitrypsin deficiency.
2 led trial of A1PI treatment in patients with alpha1 antitrypsin deficiency.
3 ding Gaucher disease, cystic fibrosis and ZZ alpha1-antitrypsin deficiency.
4 that underlies emphysema in individuals with alpha1-antitrypsin deficiency.
5 stemic inflammatory diseases associated with alpha1-antitrypsin deficiency.
6 which underlies misfolding diseases such as alpha1-antitrypsin deficiency.
7 apy for treatment of liver diseases, such as alpha1-antitrypsin deficiency.
8 tions in CF, primary ciliary dyskinesia, and alpha1-antitrypsin deficiency.
9 clinically and cost-effective therapies for alpha1-antitrypsin deficiency.
10 re, early-onset COPD probands without severe alpha1-antitrypsin deficiency.
11 a 34-year-old man with cirrhosis related to alpha1-antitrypsin deficiency.
12 n aggregation-associated diseases, including alpha1-antitrypsin deficiency.
19 ng of genetic and nongenetic modifiers in ZZ alpha1-antitrypsin deficiency and other disorders of pro
20 determine susceptibility to liver disease in alpha1-antitrypsin deficiency and provide evidence that
21 the end-stage liver disease associated with alpha1-antitrypsin deficiency and underscore the contrib
22 nically relevant PiZZ mutation, which causes alpha1-antitrypsin deficiency, and editing of phosphotyr
23 netic disorders, such as cystic fibrosis and alpha1-antitrypsin deficiency, and for other diseases, i
24 with either alcohol-related liver disease or alpha1-antitrypsin deficiency, and only one of the healt
25 he most frequent mutation that causes severe alpha1-antitrypsin deficiency arises in the SERPINA 1 ge
29 Less common causes include hemochromatosis, alpha1-antitrypsin deficiency, autoimmune hepatitis, and
31 blished pulmonary mechanics in patients with alpha1-antitrypsin deficiency, chronic obstructive pulmo
32 ess of augmentation therapy (Aug) for severe alpha1-antitrypsin deficiency, comparing strategies of:
33 sensitive measure of disease progression in alpha1 antitrypsin deficiency emphysema than spirometry
34 ents with CF, primary ciliary dyskinesia, or alpha1-antitrypsin deficiency exhibited 3-fold higher mu
35 abolic conditions studied in further detail (alpha1-antitrypsin deficiency, familial hypercholesterol
37 inhibitor (A1PI) augmentation treatment for alpha1 antitrypsin deficiency has not been substantiated
38 mphysema progression in patients with severe alpha1 antitrypsin deficiency in a randomised controlled
39 insulin signaling in the PiZ mouse model of alpha1-antitrypsin deficiency in which hepatocellular ac
45 a novel target for development of new human alpha1-antitrypsin deficiency liver disease therapies.
46 tients with alcohol-related liver disease or alpha1-antitrypsin-deficiency liver disease, and only on
48 verity and distribution in 119 subjects with alpha1-antitrypsin deficiency (PiZ phenotype) and groupe
49 ria or tuberculosis, genetic conditions (eg, alpha1-antitrypsin deficiency, primary ciliary dyskinesi
50 y centres in 13 countries if they had severe alpha1 antitrypsin deficiency (serum concentration <11 m