SAN DIEGO, Sept. 19, 2023 /PRNewswire/ -- Inhibrx, Inc. (INBX), a clinical-stage biopharmaceutical company dedicated to the development of therapeutics for oncology and rare diseases, announced today ...
(RTTNews) - Beam Therapeutics Inc. (BEAM) announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-302, a ...
Clear path forward with proposed single Phase 3 study evaluating alvelestat at the 240 mg dose level versus placebo at 12-18 months for potential full approval in both the U.S. and EU No additional ...
Acquired DNA mutations found in the SERPINA1 gene can protect liver cells from damage in patients with alpha-1 antitrypsin deficiency, new research shows. Alpha-1 antitrypsin deficiency (A1AD) is a ...
Researchers at Saint Louis University's School of Medicine, in collaboration with Arrowhead Pharmaceuticals and Takeda Pharmaceuticals, report the first effective drug to treat a rare, genetic liver ...
Study designed to demonstrate impact of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% (Alpha-1 15%), an alpha 1 antitrypsin treatment, compared with Liquid Alpha1-Proteinase Inhibitor (Human) ...
Korro Bio, Inc., a biopharmaceutical company focused on RNA editing therapies, announced that the European Medicines Agency (EMA) has granted orphan drug designation for its investigational drug ...
Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder characterized by the misfolding and accumulation of the mutant variant of alpha-1 antitrypsin (AAT) within hepatocytes, which limits its ...
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Alpha-1 antitrypsin deficiency: Study reveals why some patients with the hereditary disease remain healthy
Alpha-1-antitrypsin is a so-called protease inhibitor, a type of enzyme inhibitor. It is produced in the liver but exerts its effects in the lungs, where it regulates immune cell activity. This ...
Austin, Texas, Jan. 13, 2026 (GLOBE NEWSWIRE) -- Alpha-1 Antitrypsin Deficiency Market Size & Growth Analysis: According to SNS Insider, The Alpha-1 Antitrypsin Deficiency Market was valued at USD ...
Alpha-1 antitrypsin deficiency (AATD) is a hereditary condition characterised by the misfolding of the alpha-1 antitrypsin (AAT) protein, resulting in its hepatic accumulation and compromised protease ...
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