We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Least-squares mean percent differences in serum Z-AAT ...

David H. Perlmutter, MD, Vira I. Heinz Endowed Chairman, Department of Pediatrics, Distinguished Professor of Pediatrics, Professor of Cell Biology, University of Pittsburgh School of Medicine; ...

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 ...

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 ...

Achieved durable production of serum AAT protein at levels associated with a lower risk of AATD liver and lung diseases following repeat 200 mg doses of WVE-006 (11.9 µM total AAT, 7.2 µM M-AAT) First ...

(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 ...

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 ...

New Data Demonstrate Proportion of Corrected M-AAT Reached a Mean of 91% of Total AAT in Circulation at Day 28 Following BEAM-302 Treatment in 60 mg Cohort (n=3) Mean Decrease of 79% in Mutant Z-AAT ...

Wave Life Sciences Ltd. (NASDAQ:WVE) stock is trading higher on Wednesday after the company revealed proof-of-mechanism data from the ongoing Phase 1b/2a RestorAATion-2 study of WVE-006 in alpha-1 ...

After becoming the first group to therapeutically edit human RNA in October 2024, Wave Life Sciences has released more data from its ongoing alpha-1 antitrypsin deficiency (AATD) clinical trial—and ...

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 ...