CORDIS Project
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PRO-CF-MED aims to develop a groundbreaking treatment for cystic fibrosis patients with the F508del mutation. The project focuses on assessing the clinical efficacy of QR-010, an innovative oligonucleotide-based therapy that could transform the management of this life-threatening condition.
Cystic fibrosis (CF) is a progressive life-shortening disease caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene leading to a dysfunctional CFTR protein.
The disease affects over 70,000 patients worldwide and while many mutations are known, the F508del mutation affects 90% of all patients.
The absence of CFTR in the plasma membrane leads to a dramatic decrease in chloride efflux, resulting in viscous mucus that causes severe symptoms in vital organs like…
PROQR THERAPEUTICS NV
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Netherlands, Leiden
Type: SME
Activity type: Private for-profit entities (excluding Higher or Secondary Education Establishments)
SME: Yes
Belgium, Louvain La Neuve
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
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