We are ProQR, a biotechnology company dedicated to changing lives by developing RNA therapies for diseases with high unmet need using our proprietary Axiomer RNA-editing platform technology.
On the 10 May, 34 sporty ProQRians are taking part in the Leiden Marathon event! Each runner has selected a distance of 42, 21, 10 or 5 km to raise money for the Dutch Liver Patient Association (NLV)!
The NLV supports patients living with liver disease while also shaping research, policy and care. The liver is one of the most essential organs in the human body, performing a wide range of functions vital for life and health. Liver disease can develop for many different reasons and can affect people of any age.
Biliary atresia is a rare liver disease that affects newborns and young children. In BA, the bile ducts are blocked or absent, leading to a build-up of toxic bile in the liver. Symptoms often appear within weeks after birth, and without early treatment, the disease can rapidly progress to liver failure. Even with surgery, many children will eventually require a liver transplant. At ProQR, we are developing AX-0810, a potential RNA-editing therapy for the treatment of biliary atresia.
Through our participation, we are proud to support the NLV in their mission to improve the lives of people affected by liver disease and to drive progress in research and care.