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; ...

Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 antitrypsin deficiency - a promising leap toward treating humans with this rare ...

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 ...

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 ...

(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 ...

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 ...

Chronic obstructive pulmonary disease (COPD) remains highly prevalent among veterans, but the contribution of alpha-1 antitrypsin deficiency (AAT deficiency or AATD) to this patient population remains ...

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 ...

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 ...

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 ...