Krystal Biotech Announces Promising Clinical Data for KB408 Gene Therapy in Alpha-1 Antitrypsin Deficiency Patients and Progress on KB407 for Cystic Fibrosis December 12, 2024 — 08:13 am EST ...

SAN DIEGO — After bronchoscopic lung volume reduction, or BLVR, patients with COPD and alpha-1 antitrypsin deficiency had better lung function, according to a poster at the American Thoracic ...

About Alpha-1 Antitrypsin Deficiency (AATD) AATD is an inherited genetic disorder that can cause early onset emphysema and liver disease.

Gastroenterology (2024). [2] Selection for somatic escape variants in SERPINA1 in the liver of patients with alpha-1 antitrypsin deficiency. Nature Genetics (2025).

Mohammad, N., et al. (2024). Liver Characterization of a Cohort of Alpha-1 Antitrypsin Deficiency Patients with and without Lung Disease. Journal of Clinical and Translational Hepatology. doi.org ...

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In a dose-dependent manner, fazirsiran reduced serum and liver concentrations of Z-AAT and also improved histological measures in patients with homozygous ZZ alpha-1 antitrypsin deficiency ...

Congress can modernize our healthcare system by passing the SOAR Act and enabling beneficiaries to live on their terms with ...

The liver disease caused by alpha-1-antitrypsin deficiency (ATD) is predominantly characterized by fibrosis/cirrhosis with relatively limited inflammation and an increased likelihood of hepatic ...

News GlobeNewswire Beam Therapeutics Presents Additional Data for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) at 2025 Alpha-1 Foundation 7th Global Research Conference and 10th Patient Congress ...