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Review
. 2023 Sep:47:23-26.
doi: 10.1016/j.prrv.2023.06.001. Epub 2023 Jun 17.

An innovative strategy for personalised medicine in a CFSPID case that evolved with time

J Twynam-Perkins  1 , A Fall  2 , J W Lefferts  3 , D S Urquhart  4
Affiliations
Review

An innovative strategy for personalised medicine in a CFSPID case that evolved with time

J Twynam-Perkins et al. Paediatr Respir Rev. 2023 Sep.

Abstract

We present a challenging case that illustrates how the clinical manifestations in children with CFTR mutations of uncertain significance may change over time. This case highlights the evolution of confirming a diagnosis of CF and emphasises the importance of regular review and monitoring of this patient cohort.

Keywords: CFTR modulators; N = 1 clinical trials; Organoids.

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Conflict of interest statement

Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

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