Patients Creating new possibilities for patients
At ProQR, our mission is to edit RNA, treat disease and change lives. This guides every decision we make as we work to develop new treatment options for people living with high unmet need.
Our vision is to create a world where genetics informs your health - but never limit it. By harnessing the power of RNA editing, we strive to make our vision for the future a reality.
Biliary atresia
Biliary atresia (BA) is a rare liver disease that affects newborns and young children.
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Hurler syndrome
Hurler syndrome is the most severe form of mucopolysaccharidosis type I (MPS I), a rare inherited lysosomal storage disease.
Internal link Learn more about Hurler syndrome