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J Cyst Fibros. 2019 Sep;18(5):685-692. doi: 10.1016/j.jcf.2019.06.010. Epub 2019 Jul 17.

Theranostics by testing CFTR modulators in patient-derived materials: The current status and a proposal for subjects with rare CFTR mutations.

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University of Lisboa, Portugal.
National Heart & Lung Institute, Imperial College London, Royal Brompton & Harefield NHS Foundation Trust, London, UK. Electronic address:
University of Leuven, Belgium.
Motol University Hospital Prague, Czech Republic.
Queens University, Belfast, UK.
Hadassah Hebrew University Medical Center, Jerusalem, Israel.
Leeds Teaching Hospitals NHS Trust, Leeds, UK.


The last decade has witnessed developments in the CF drug pipeline which are both exciting and unprecedented, bringing with them previously unconsidered challenges. The Task Force group came together to consider these challenges and possible strategies to address them. Over the last 18 months, we have discussed internally and gathered views from a broad range of individuals representing patient organizations, clinical and research teams, the pharmaceutical industry and regulatory agencies. In this and the accompanying article, we discuss two main areas of focus: i) optimising trial design and delivery for speed/efficiency; ii) drug development for patients with rare CFTR mutations. We propose some strategies to tackle the challenges ahead and highlight areas where further thought is needed. We see this as the start of a process rather than the end and hope herewith to engage the wider community in seeking solutions to improved treatments for all patients with CF.


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