Dicerna receives orphan drug designation from FDA for DCR-A1AT to treat alpha-1 antitrypsin deficiency

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A1AT deficiency is a genetic disorder that can cause lung and liver disease. Children with the liver manifestations of the disease may present with yellowing of the skin

The post Dicerna receives orphan drug designation from FDA for DCR-A1AT to treat alpha-1 antitrypsin deficiency appeared first on Pharmaceutical Business review.

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