Inhaled alpha-1-antitrypsin (AAT) restores lower respiratory tract protease- antiprotease homeostasis and reduces inflammation in AAT-deficient individuals: A randomized phase 2 study 

Mark Brantly1, James Stocks2, Jorge Lascano1, Tammy Flagg1, Ann M. Jeffers2, Shuzi Z. Owens2, Torry A. Tucker2, Megan Devine2 Noga […]

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C-Path Welcomes New Advisory Membersto Alpha-1 Antitrypsin Deficiency Consortium

Critical Path Institute’s (C-Path) Critical Path for Alpha-1 Antitrypsin Deficiency (CPA-1) consortium today announced the addition of several key advisory […]

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Biomarkers Associated with Future Severe Liver Disease in Children with Alpha-1 Antitrypsin Deficiency

“For many years we have known that most kids with ZZ Alpha-1 do fine, but a few get very sick […]

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Kelly, Tillis Introduce Bipartisan Legislation to Increase Access to Plasma-Based Medicines

Today, Senators Mark Kelly (D-AZ) and Thom Tillis (R-NC) introduced the bipartisan Preserving Life-saving Access to Specialty Medicines in America […]

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Verona Pharmaceuticals announces FDA approval for Ensifentrine (Ohtuvayre), a treatment for COPD

On June 26, 2024, Verona Pharmaceuticals announced that its new medication, Ensifentrine (Ohtuvayre), was approved by the U.S. Food and […]

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Alpha-1 Foundation and 130 Organizations Support 2024 Reauthorization of the Rare Pediatric Disease Priority Review Voucher Program

Dear Senate Majority Leader Chuck Schumer, Senate Minority Leader McConnell, Chairman Sanders, and Ranking Member Cassidy,  On behalf of the […]

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Saint Louis University Researchers Identify a Biomarker for Severe Liver Disease in Infants

ST. LOUIS — In a new study, Saint Louis University School of Medicine researchers and colleagues have identified biomarkers in […]

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