NEW YORK (GenomeWeb) – Arrowhead Research announced today that it has filed an application with regulators to begin a Phase I trial of its alpha-1 antitrypsin deficiency (AAT) drug ARC-AAT in Australia.

AAT deficiency is a genetic disease that leads to decreased activity of the protease inhibitor alpha-1 antitrypsin in the blood and lungs causing lung dysfunction. It can also feature the accumulation of a mutant AAT protein known as Z-AAT in liver tissue, which leads to liver injury, fibrosis, cirrhosis, and potentially liver cancer.

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Cancer researcher Alan Rabson has died at 92, the New York Times reports.

As the National Guideline Clearinghouse goes dark, the ECRI Institute says it will pick up the slack.

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Jul
19
Sponsored by
Thermo Fisher Scientific

This webinar will discuss how ultra-highly sensitive and customizable targeted next-generation sequencing panels are applied in inherited disease research. 

Aug
07
Sponsored by
Qiagen

This webinar will present the results of an evaluation of a web-based variant interpretation software system for clinical next-generation sequencing.