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

Source link

Related posts

Sero-prevalence of Helicobacter pylori CagA immunoglobulin G antibody, serum pepsinogens and haemoglobin levels in adults


A recurrent cancer-associated substitution in DNA polymerase ε produces a hyperactive enzyme


Slow Medicine: Fecal Cancer Screen; Illness Isn't About 'Fighting'


This website uses cookies to improve your experience. We'll assume you're ok with this, but you can opt-out if you wish. Accept Read More

Privacy & Cookies Policy