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

Deliverables

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
Publisher: poster at the North American Cystic Fibrosis Conference

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
Publisher: Dove Medical Press Ltd
DOI: 10.2147/CPAA.S100759

Intellectual Property Rights

OLIGONUCLEOTIDES FOR MAKING A CHANGE IN THE SEQUENCE OF A TARGET RNA MOLECULE PRESENT IN A LIVING CELL

Application/Publication number: EP 13760133
Date: 2013-07-12
Applicant(s): PROQR THERAPEUTICS NV

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