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Clinical Proof of concept for a RNA-targeting Oligonucleotide for a Cystic fibrosis-F508del MEDication

Deliverables

PRO-CF-MED project website developed including project updates and patient ‘friendly’ section

PRO-CF-MED project website developed including project updates and patient ‘friendly’ section

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Publications

QR-010 significantly improves CFTR function in nasal potential difference proof of concept study in subjects with CF homozygous for the F508del-CFTR mutation

Author(s): I Sermet-Gaudelus, D Nichols, JA Nick, C De Boeck, JP Clancy, GM Solomon, SM Rowe, JS Elborn, MA MAll, JA Bolognese, F Bouisset, W den Hollander, N Lamontagne, N Tomkinson, N Henig
Published in: 2016

Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives

Author(s): Sabrina Noel, Bela Z Schmidt, Jeremy Haaf, Teresinha Leal
Published in: Clinical Pharmacology: Advances and Applications, Issue Volume 8, 2016, Page(s) 127-140, ISSN 1179-1438
DOI: 10.2147/CPAA.S100759