Trial underway for liver disease-causing genetic disorder treatment

The European Commission granted orphan drug designation to Dicerna Pharmaceuticals’ DCR-A1AT for the treatment of congenital alpha-1 antitrypsin deficiency, a genetic disorder that can lead to liver disease in children and adults, according to a press release.
“We are gratified to see regulators recognize the urgent need for the development of a safe and effective therapy for treatment of alpha-1 antitrypsin deficiency,” Ralf Rosskamp, MD, chief medical officer of Dicerna, said in the release. “This orphan drug designation from the European Commission acknowledges the

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