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Drug Res (Stuttg) 2018; 68(S 01): S21-S23
DOI: 10.1055/a-0733-0883
Symposium der Paul-Martini-Stiftung
Georg Thieme Verlag KG Stuttgart · New York

Disease-Specific Clinical Trial Networks: the Example of Cystic Fibrosis

Silke van Koningsbruggen-Rietschel
Mukoviszidose-Zentrum Köln, Pädiatrische Pneumologie und Allergologie, Klinik und Poliklinik für Kinder- und Jugendmedizin, Universität zu Köln
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Publication Date:
19 November 2018 (online)

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Cystic Fibrosis (CF) is an autosomal recessive, life-shortening multiorgan disorder. It is the most frequent rare disease affecting about 42.000 patients in Europe and more than 80.000 patients worldwide. CF is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene leading to absent or defective chloride channels (CFTR).

 

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