Article
Advancing the pipeline of cystic fibrosis clinical trials: a new roadmap with a global trial network perspective.
The Lancet. Respiratory medicine - 1 Oct 2023
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
Abstract excerpt
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 divided by CFTR modulator eligibility. The development of a robust pipeline of nucleic acid-based therapies...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.