Data from the RestorAATion-2 clinical trial of WVE-006 (GalNAc-RNA editing), including new data from the 400 mg multidose cohort and 600 mg ...

Alpha-1 Antitrypsin Deficiency (AATD) is a rare genetic disorder caused by SERPINA1 gene mutations, leading to conditions like pulmonary emphysema and liver dysfunction. The AATD market is in its ...

Patients with lung disease are more likely to develop heart disease independent of any risk factors, according to new research. Boosting levels of a deficient protein has clear survival benefits for ...

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 ...

MOLINE, Ill. — November is Alpha-1 Awareness Month, a time dedicated to increasing understanding of Alpha-1 Antitrypsin Deficiency, a rare genetic condition that can cause serious lung and liver ...

BEAM-302 demonstrated safety and efficacy in increasing AAT levels and reducing mutant protein in alpha-1 antitrypsin deficiency patients. Beam Therapeutics announced promising initial results from ...

Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder associated with liver disease, ranging from fibrosis to hepatocellular carcinoma. The disease remains asymptomatic until its final stages ...

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 ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Studies assessing bronchoscopic lung volume reduction ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Least-squares mean percent differences in serum Z-AAT ...

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 ...