Perspective: A test for those left behind

Nearly a decade on from the introduction of the first CFTR modulator, these therapies have led to reduced levels of organ damage and chronic infection, while improving lung function, exercise capacity, nutritional status, quality of life and even survival. Furthermore, the standard measurement of lu...

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Bibliographic Details
Published in:Nature (London) 2020-07, Vol.583 (7818), p.S5-S5
Main Author: Rowe, Steven M
Format: Article
Language:English
Subjects:
Chloride
Chronic infection
Cystic fibrosis
Drugs
FDA approval
Lungs
Mutation
Nutritional status
Pathology
Quality of life
Respiratory function
Salty taste
Sweat
Sweat gland
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Summary:Nearly a decade on from the introduction of the first CFTR modulator, these therapies have led to reduced levels of organ damage and chronic infection, while improving lung function, exercise capacity, nutritional status, quality of life and even survival. Furthermore, the standard measurement of lung function using spirometry (the FEV1 test) is ineffective at detecting improvement in the increasing number of people with cystic fibrosis who have high lung function. The absence of CFTR results in high chloride levels, a characteristic often first recognized by a salty taste on the skin. Because the sweat gland is not affected by progressive pathology, its reading of CFTR function remains accurate. The scientific community must rapidly adapt to these circumstances to maintain the marked pace of therapeutic research. "Because the sweat gland is not affected by progressive pathology, its reading of CFTR function remains accurate 1.
ISSN:0028-0836
1476-4687
DOI:10.1038/d41586-020-02107-9