Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
8813014 | Paediatric Respiratory Reviews | 2018 | 7 Pages |
Abstract
Whilst substantial progress has been made in the treatment of cystic fibrosis, the disease still carries a significant burden in terms of symptoms, requirement for treatment and early mortality. The last decade has witnessed a new era in the development of small molecule drugs targeting the CFTR protein, which for the first time may provide a truly disease-modifying approach to treatment. This article reviews progress and highlights some of the current and future challenges in CFTR modulator therapies.
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Authors
Katharine Harman, Rebecca Dobra, Jane C. Davies,