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Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency

• Authors : Arriva Pharmaceuticals, Inc.

• Source: A Phase 1B/2A Study to Evaluate the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects

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Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency

• Authors : Arriva Pharmaceuticals, Inc.

• Source: Phase I Safety Investigation of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha

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Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency

• Authors : National Heart, Lung, and Blood Institute (NHLBI); Alpha-1 Foundation; University of Florida

• Source: Preclinical & Phase I/II Trials of AAV-AAT Vectors: Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Alpha

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Study of Genotype and Phenotype in Patients With Alpha 1-Antitrypsin Deficiency

• Authors : University of Pittsburgh

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Study of BMN 349 Single Dose in (PiZZ) and (PiMZ) Adult Participants (PiZZ)

• Source: A Randomized, Double-Blind, Placebo-Controlled, Single Oral Dose Study Evaluating the Safety and Pharmacokinetics of BMN 349 in Homozygous for the Z Mutation of Alpha 1

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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

• Authors : Takeda Development Center Americas, Inc.

• Source: A Randomized, Double-blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Fazirsiran in the Treatment of Alpha-1 Antitrypsin

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A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency (Serpentine-1)

• Source: A Phase 1 Study of Inhaled KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency

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Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease (A1BC)

• Authors : Alpha-1 Foundation; National Heart, Lung, and Blood Institute (NHLBI); Jeanine D'Armiento, Associate Professor of Medicine (in Anesthesiology)

• Source: Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease - Alpha-1 Biomarker Research Consortium (A1BC)

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PiMZ Longitudinal Cohort (PiMZ Logic)

• Authors : Alpha-1 Foundation; Monica Goldklang, Associate Professor of Medicine (in Anesthesiology)

• Source: Alpha-1 Antitrypsin PiMZ Longitudinal Cohort (PiMZ Logic)

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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)

• Source: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Safety and Efficacy of Fazirsiran in the Treatment of Alpha-1 Antitrypsin

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