Objective: To evaluate the prevalence of alpha-1 antitrypsin (AAT) variants through SERPINA1 genotyping in patients with non-cystic fibrosis bronchiectasis, and assess their clinical, functional and ...
In her early 30s, Bonnie began to experience frequent shortness of breath. Though she was still active, she noticed that everyday tasks like exercising, climbing stairs and dancing left her breathless ...
Additional orphan designation reinforces Sanofi’s commitment to developing treatments for rare diseases Efdoralprin alfa, an investigational restorative recombinant therapy, recently met all primary ...
Objective: Severe Pi*ZZ alpha-1 antitrypsin (AAT) deficiency, caused by the Glu342Lys mutation in the SERPINA1 gene, resulting in protein misfolding and polymerization in hepatocytes, and proteotoxic ...
ALPHA DRIVE ONE will soon be starring on their very first reality show! On December 9, Mnet announced that the original reality show “ALPHA DRIVE ONE, Let’s Go,” which captures the pre-debut journey ...
Regeneron is adding its name to Tessera Therapeutics’ unique in vivo gene-writing program, inking a deal that lets it in on a prospect for alpha-1 antitrypsin deficiency (AATD) through a $150 million ...
Regeneron Pharmaceuticals is putting more money into gene editing, announcing Monday a partnership with Tessera Therapeutics to develop an experimental program for a rare liver and lung disease. At ...
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