Antitrypsin Deficiency

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 Society International Conference.

Wave Life Sciences has announced positive results from its phase 1b/2a clinical trial of WVE-003, a potential disease-modifying therapeutic for Huntington’s disease.

Aortic stiffness, a known risk factor for cardiovascular disease, was raised among patients with alpha-1 antitrypsin deficiency vs. patients with COPD or healthy individuals, according to study results.

Patients with severe alpha-1 antitrypsin deficiency had improved survival with intravenous plasma-purified alpha-1 antitrypsin therapy, according to results published in American Journal of Respiratory and Critical Care Medicine.

The FDA granted orphan drug designation for KB408 for the treatment of alpha-1 antitrypsin deficiency, according to a press release from Krystal Biotech.

Arrowhead Pharmaceuticals announced that the first patient has been treated in Takeda Pharmaceuticals’ phase 3 clinical trial of fazirsiran for the treatment of alpha-1 antitrypsin deficiency associated liver disease.

WASHINGTON — Elevated Z polymer levels may predict lung and liver disease activity in patients with alpha 1 antitrypsin deficiency, according to a presenter at The Liver Meeting.

Direct-to-consumer testing, combined with clinical follow-up, helped identify patients with undiagnosed alpha-1 antitrypsin deficiency, according to data published in Chest.

Using a novel RNA interference therapeutic improved fibrosis and reduced toxic proteins and globular burden in alpha-1 antitrypsin deficiency, according to a presenter at the International Liver Congress.