title: Progress in cystic fibrosis and the CF Therapeutics Development Network.
abstract: Cystic fibrosis (CF), the most common life-shortening genetic disorder in Caucasians, affects approximately 70 000 individuals worldwide. In 1998, the Cystic Fibrosis Foundation (CFF) launched the CF Therapeutics Development Network (CF-TDN) as a central element of its Therapeutics Development Programme. Designed to accelerate the clinical evaluation of new therapies needed to fulfil the CFF mission to control and cure CF, the CF-TDN has conducted 75 clinical trials since its inception, and has contributed to studies as varied as initial safety and proof of concept trials to pivotal programmes required for regulatory approval. This review highlights recent and significant research efforts of the CF-TDN, including a summary of contributions to studies involving CF transmembrane conductance regulator (CFTR) modulators, airway surface liquid hydrators and mucus modifiers, anti-infectives, anti-inflammatories, and nutritional therapies. Efforts to advance CF biomarkers, necessary to accelerate the therapeutic goals of the network, are also summarised.
authors.affiliation: Department of Medicine, University of Alabama at Birmingham, 1819 University Boulevard (MCLM 768), Birmingham, AL 35294, USA. smrowe@uab.edu
authors.forename: Steven M
authors.initials: SM
authors.lastname: Rowe
authors.forename: Drucy S
authors.initials: DS
authors.lastname: Borowitz
authors.forename: Jane L
authors.initials: JL
authors.lastname: Burns
authors.forename: John P
authors.initials: JP
authors.lastname: Clancy
authors.forename: Scott H
authors.initials: SH
authors.lastname: Donaldson
authors.forename: George
authors.initials: G
authors.lastname: Retsch-Bogart
authors.forename: Scott D
authors.initials: SD
authors.lastname: Sagel
authors.forename: Bonnie W
authors.initials: BW
authors.lastname: Ramsey
mesh_terms: D035843:Biomedical Research / Q000639:trends*; D002986:Clinical Trials as Topic / Q000639:trends*; D003195:Computer Communication Networks / Q000458:organization & administration; D003550:Cystic Fibrosis / Q000628:therapy*; D006801:Humans; D007400:Interprofessional Relations; D012107:Research Design; D014481:United States