Universität zu Köln

van Koningsbruggen-Rietschel, S. and Rietschel, E. (2017). Cystic fibrosis. Disease on the way to personalized therapy. Mon.schr. Kinderheilkd., 165 (8). S. 681 - 688. NEW YORK: SPRINGER. ISSN 1433-0474

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Abstract

By the introduction of newborn screening it has become possible in most patients to implement symptomatic therapy for cystic fibrosis in the first weeks of life. The life expectancy and the quality of life of patients and their families will be improved by this implementation. Many mutation-specific therapies as well as mutation-agnostic treatments are in preclinical and clinical development and two have already been approved. Because of the difficulties in evaluating the individual effectiveness of these therapies by using clinical parameters alone, measurement of improved chloride channel function in vivo and in vitro is becoming more important.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code van Koningsbruggen-Rietschel, S. UNSPECIFIED UNSPECIFIED UNSPECIFIED Rietschel, E. UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-223261
DOI:	10.1007/s00112-017-0322-0
Journal or Publication Title:	Mon.schr. Kinderheilkd.
Volume:	165
Number:	8
Page Range:	S. 681 - 688
Date:	2017
Publisher:	SPRINGER
Place of Publication:	NEW YORK
ISSN:	1433-0474
Language:	German
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language DOUBLE-BLIND; IVACAFTOR Multiple languages Pediatrics Multiple languages
Refereed:	Yes
URI:	http://kups.ub.uni-koeln.de/id/eprint/22326