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Progress in cystic fibrosis and the CF Therapeutics Development Network
  1. Steven M Rowe1,
  2. Drucy S Borowitz2,
  3. Jane L Burns3,
  4. John P Clancy4,
  5. Scott H Donaldson5,
  6. George Retsch-Bogart5,
  7. Scott D Sagel6,
  8. Bonnie W Ramsey3
  1. 1Department of Medicine, University of Alabama at Birmingham, Birmingham, Alabama, USA
  2. 2Department of Pediatrics, State University of New York at Buffalo, Buffalo, New York, USA
  3. 3Seattle Children's Hospital and the University of Washington, Seattle, Washington, USA
  4. 4Department of Pediatrics, Cincinnati Children's Hospital Medical Center, and the University of Cincinnati, Cincinnati, Ohio, USA
  5. 5University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA
  6. 6Department of Pediatrics, Children's Hospital Colorado and University of Colorado, Aurora, Colorado, USA
  1. Correspondence to Dr Steven M Rowe, University of Alabama at Birmingham, 1819 University Boulevard (MCLM 768), Birmingham, AL 35294, USA; smrowe{at}uab.edu

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.

  • Cystic Fibrosis

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