News and Trends 30 Nov 2017
RNA Therapy for Rare Skin Disease Gets Orphan Status
The EMA has granted orphan drug designation to ProQR’s QR-313, a possible first disease-modifying treatment for the rare disease dystrophic epidermolysis bullosa. Dystrophic epidermolysis bullosa (DEB) is a genetic disease that causes the skin to easily blister, severely affecting the quality of life of patients since a very early age. Current treatment is limited to […]