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Alpha-1 antitrypsin deficiency

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Search Period All yearsLast 12 monthsLast 5 years Topic All topicsGeneralNutrition and dietAlternative treatment methods Type of research Any type of researchHighest Level of Evidence Only (35)Meta-analysis (8)Treatment guideline (5)Phase 3 study (8)Randomized trial (8)Systematic review (6)Clinical Trial (169)Review article (176)Early study (phase 1 or 2) (18)Case report (114)Laboratory or animal study (26)Commentary or editorial (90)Retracted publication (1) Only studies currently recruiting participants (28)

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Publications and studies (1172)

• Quantitative Analysis of Bronchiectasis in Alpha-1 Antitrypsin Deficiency. (2026/01/01) ♡
• Phase 3 studyiResearch in a large group of patients, the final step before a treatment can be approved. What comes out here carries significant weight. A Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD) (2026-08-11) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD) (2026-08-10) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems (2026-07-24) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD) (2026-07-24) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Development of a Prediction Score for the Occurrence of Death or Lung Transplantation in Patients With Emphysema Secondary to Alpha-1-anti-tripsin Deficiency (2026-07-20) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Role of Genetic Factors in the Development of Lung Disease (2026-07-17) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. IBS-Oriented CDED Versus Low FODMAP Diet for Irritable Bowel Syndrome (2026-07-16) ♡
• Phase 3 studyiResearch in a large group of patients, the final step before a treatment can be approved. What comes out here carries significant weight. Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD) (2026-06-23) ♡
• Phase 3 studyiResearch in a large group of patients, the final step before a treatment can be approved. What comes out here carries significant weight. Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency (2026-06-23) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Natural Course and Molecular Basis of Alpha 1- Antitrypsin Deficiency-associated Liver Disease. (2026-06-10) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC) (2026-06-04) ♡
• Phase 3 studyiResearch in a large group of patients, the final step before a treatment can be approved. What comes out here carries significant weight. Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza / Zemaira Weekly for 3 Years in Adults With alpha1 Antitrypsin Deficiency (AATD) (2026-05-15) ♡
• Phase 3 studyiResearch in a large group of patients, the final step before a treatment can be approved. What comes out here carries significant weight. Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein (2026-05-13) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants (2026-05-06) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Alpha-1 Antitrypsin Deficiency Adult Liver Study (2026-04-27) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. A Phase 1/2a, Single- and Multiple-dose Escalation Study of KRRO-110 (2026-04-17) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD (2026-04-14) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema (2026-04-13) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. A Study of Belcesiran in Patients With AATLD (2026-04-07) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1) (2026-03-23) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) (2026-03-20) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. Gene Therapy for Alpha 1- Antitrypsin Deficiency (2026-03-13) ♡
• Phase 3 studyiResearch in a large group of patients, the final step before a treatment can be approved. What comes out here carries significant weight. Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis) (2026-03-05) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. PiMZ Longitudinal Cohort (PiMZ Logic) (2026-01-16) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease (2026-01-16) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Alpha-1 Foundation DNA and Tissue Bank (2026-01-15) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD) (2026-01-12) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Prevalence of Alpha-1 Antitrypsin Deficiency in Non-Cirrhotic Liver Cancer (2026-01-05) ♡
• Clinical features in patients with severe Alpha-1 antitrypsin deficiency due to rare genotypes. (2025/12/31) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Chronic Obstructive Pulmonary Disease in Never-Smokers-A Distinct Entity Within the COPD Spectrum. (2025/12/26) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Non-tuberculous mycobacteria (NTM) and COPD: a multicentre prospective study. (2025/12/21) ♡
• Randomized researchiParticipants were divided into groups by lottery and compared with each other. This reduces the chance that a difference is due to something other than the treatment. Two randomised controlled phase 2 studies of the oral neutrophil elastase inhibitor alvelestat in alpha-1 antitrypsin deficiency. (2025/12/18) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Alvelestat for alpha-1 antitrypsin deficiency-associated emphysema: the hope for an oral treatment. (2025/12/18) ♡
• Pulmonary rehabilitation and quality of life in alpha-1 antitrypsin deficiency: findings from a retrospective cohort study. (2025/12/15) ♡
• Circulating C-terminal peptides and polymers of alpha-1 antitrypsin as putative markers of pediatric Pi*ZZ liver disease. (2025/12/10) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Searching for alpha-1 antitrypsin deficiency in patients with bronchiectasis: reducing idiopathic cases. (2025/12/08) ♡
• Prevalence of SERPINA1 mutations in a bronchiectasis cohort: implications of extended screening for alpha-1 antitrypsin deficiency. (2025/12/05) ♡
• Performance of enhanced liver fibrosis test and indirect serum fibrosis markers for exclusion of advanced liver fibrosis in alpha-1 antitrypsin deficiency. (2025/12/01) ♡
• Growth hormone resistance in children with chronic cholestatic liver disease and reduced skeletal muscle mass. (2025/12/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Seasonal Changes in Circulating Alpha-1 Antitrypsin Are Altered by Sex and Genetic Deficiency. (2025/12/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Inflammation-adjusted optimal serum cut-off alpha-1 antitrypsin values for detecting deficient patients: a cross-sectional analysis of the AVATAR cohort. (2025/12/01) ♡
• Neutrophil-derived extracellular vesicles in the plasma of alpha-1 antitrypsin deficient individuals reveal pro-inflammatory metabolic and transcriptomic signatures. (2025/12/01) ♡
• Systematic reviewiAll research on one question is searched according to fixed rules and compared side by side, so that no studies with poor outcomes are missed. Patient-reported outcome measures for use in patients with alpha-1-antitrypsin deficiency: results of a systematic selection process. (2025/11/27) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. The phenotypic state of biallelic SERPINA1 Q0 variants in α(1)-antitrypsin deficiency. (2025/11/24) ♡
• Characterization of a novel SERPINA1 variant carrying two missense mutations: molecular mechanisms and functional impact. (2025/11/21) ♡
• Molecular insights on the mechanism of α1-antitrypsin condensate formation and maturation. (2025/11/19) ♡
• The mechanism of pathogenic α(1)-antitrypsin aggregation in the human liver. (2025/11/18) ♡
• Matrix metalloproteinases and disease severity among PI*MZ and PI*ZZ individuals in the Genomic Research in Alpha-1 Antitrypsin Deficiency and Sarcoidosis (GRADS) cohort. (2025/11/17) ♡
• Quantitative analysis of chest computed tomography in alpha-1-antitrypsin deficiency. (2025/11/15) ♡

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