Advancing the pipeline of cystic fibrosis clinical trials: a new roadmap with a global trial network perspective

The growing use of modulator therapies aimed at restoring cystic fibrosis transmembrane conductance regulator (CFTR) protein function in people with cystic fibrosis has fundamentally altered clinical trial strategies needed to advance new therapeutics across an orphan disease population that is now...

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Veröffentlicht in:	The lancet respiratory medicine 2023-10, Vol.11 (10), p.932-944
Hauptverfasser:	Mayer-Hamblett, Nicole, Clancy, John Paul, Jain, Raksha, Donaldson, Scott H, Fajac, Isabelle, Goss, Christopher H, Polineni, Deepika, Ratjen, Felix, Quon, Bradley S, Zemanick, Edith T, Bell, Scott C, Davies, Jane C, Jain, Manu, Konstan, Michael W, Kerper, Natanya R, LaRosa, Tré, Mall, Marcus A, McKone, Edward, Pearson, Kelsie, Pilewski, Joseph M, Quittell, Lynne, Rayment, Jonathan H, Rowe, Steven M, Taylor-Cousar, Jennifer L, Retsch-Bogart, George, Downey, Damian G
Format:	Artikel
Sprache:	eng
Schlagworte:	Cystic Fibrosis - drug therapy Cystic Fibrosis Transmembrane Conductance Regulator - genetics Genetic Therapy Humans Mutation Quality of Life
Online-Zugang:	Volltext
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