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.

One of our focus areas is Rett Syndrome. Rett syndrome is a rare neurodevelopmental disorder that is caused, in most cases, by a spontaneous mutation in the MECP2 gene, which encodes Methyl-CpG-binding Protein 2. It occurs in about 1 in 10 – 12,000 births in girls and is even rarer in boys. In the Netherlands, more than 250 girls are currently known to have Rett syndrome. The disease leads to severe impairments, affecting nearly every aspect of the child’s life including their ability to speak, walk, eat, and breathe.

De Nederlandse Rett Syndroom Vereniging is a patient advocacy group dedicated to supporting Rett syndrome patients and their families. In 2024, multiple ProQRians signed up to run 5, 10, 21 or 42 km as part of the Leiden Marathon event to raise money for De Nederlandse Rett Syndroom Vereniging. In total, the runners raised €3,375!

This year, over 30 ProQRians have signed up to take part in the Leiden Marathon event! The runners will again be raising money for De Nederlandse Rett Syndroom Vereniging to support them in their important work.

If you are able, please consider donating to support the runners - and more importantly to raise funds for De Nederlandse Rett Syndroom Vereniging!