Effects of Lumacaftor/Ivacaftor on Cystic Fibrosis Disease Progression in Children 2 through 5 Years of Age Homozygous for <i>F508del-CFTR</i>: A Phase 2 Placebo-controlled Clinical Trial.

"Author disclosures are available with the text of this article at www.atsjournals.org."

"AcknowledgmentThe authors thank the children and their families for participating in this trial and the trial investigators and coordinators for their contributions to the trial. Medical writing and editorial support were provided by Nathan Blow, Ph.D., of Vertex Pharmaceuticals Incorporated, under the direction of the authors. Editorial coordination and support were provided by Thomas Pickette, Pharm.D., M.B.A., and Emily Poulin, Ph.D., of Vertex Pharmaceuticals Incorporated and by Linda Gorman, Ph.D., of the Envision Pharma Group, contracted by Vertex Pharmaceuticals Incorporated. Nathan Blow, Thomas Pickette, and Emily Poulin may own stock or stock options in Vertex Pharmaceuticals Incorporated. Linda Gorman may own stock or stock options in Envision Pharma Group. The authors acknowledge the contributions of Anita Maniktala, M.D., of ICON plc Global Strategic Solutions and Vertex Pharmaceuticals Incorporated, who served as medical monitor for this study. Anita Maniktala may own stock or stock options in those companies. Christopher Edwards, Ph.D., C.M.P.P., and Karen Kaluza Smith, Ph.D., C.M.P.P., provided medical writing and editorial support on an early draft of the manuscript under the direction of the authors. Christopher Edwards and Karen Kaluza Smith are employees of ArticulateScience, LLC, which received funding from Vertex Pharmaceuticals Incorporated. Supported by Vertex Pharmaceuticals Incorporated."

"Conclusions: This placebo-controlled study suggests the potential for early disease modification with LUM/IVA treatment, including that assessed by chest MRI, in children as young as 2 years of age.: Clinical trial registered with www.clinicaltrials.gov (NCT 03625466)."