Supported by Vertex Pharmaceuticals, Cystic Fibrosis Foundation Therapeutics, and grants from the Food and Drug Administration Office of Orphan Products Development (FD-R-003432-01), the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health (NIH) (K23 DK075788, to Dr. Rowe), Cystic Fibrosis Foundation Therapeutics (CLANCY05Y2, to Dr. Clancy and the University of Alabama at Birmingham for coordinating the nasal potential difference qualification process; Clinical Facilitation Award DURIE06A0, to Dr. Durie; and RAMSEY03Y0, for the Therapeutics Development Network Coordinating Center, to Dr. Ramsey), the National Center for Research Resources of the NIH (GCRC UL1 RR025005, to Johns Hopkins; GCRC M01 RR00070 and CTSA 1UL1 RR025744, to Stanford University; UL1 RR024989, to Case Western Reserve University; GCRC M01 RR02172, to Children’s Hospital Boston; CTSA UL1 RR025014, to the Institute of Translational Health Sciences and the University of Washington; K23 RR018611-05, to Dr. Sagel; CTSA UL1 RR025780, to the University of Colorado Denver; CTSA UL 1RR025747, to the University of North Carolina at Chapel Hill; CTSA UL1 RR024153, to Children’s Hospital of Pittsburgh; and CTSA UL1 RR024134, to Children’s Hospital of Philadelphia), and the National Heart, Lung, and Blood Institute of the NIH (U01 HL081335, to the University of Colorado Denver).
Dr. Accurso reports receiving consulting fees from Inspire and PTC Therapeutics, grant support from Digestive Care, PTC Therapeutics, Gilead, Vertex, Altus, KaloBios, Axcan Pharma, Yasoo Health, INO Therapeutics, Mpex, and Genentech, and travel support from Vertex Pharmaceuticals, PTC Therapeutics, Inspire Pharmaceuticals, and Gilead Sciences; Dr. Rowe, receiving grant support from Vertex, Novartis, and PTC Therapeutics and consulting fees from PTC Therapeutics and Vertex; Dr. Clancy, receiving consulting fees from Transave and grant support from Vertex, MedImmune, Inspire, Gilead, Transave, and PTC Therapeutics; Dr. Boyle, receiving consulting fees from Novartis, Genentech, Gilead, and Bayer and grant support from Vertex; Dr. Dunitz, receiving grant support from Inspire, Vertex, Altus, Gilead, and Roche (through a clinical trial subcontract with Duke) and travel support from Vertex; Dr. Durie, receiving grant support from Vertex and consulting fees and travel support from Vertex; Dr. Sagel, receiving grant support from Vertex, Yasoo Health, and INO Therapeutics; Dr. Donaldson, receiving consulting fees from Liquidia Technologies, grant support from Inspire, Vertex, and Gilead, and travel support from Gilead and Vertex; Dr. Hornick, receiving consulting fees from Pfizer and Genentech, speaking fees from Pfizer and Boehringer Ingelheim, and travel support from Pfizer, Boehringer Ingelheim, and Genentech; Dr. Konstan, receiving grant support from Vertex, Novartis, Boehringer Ingelheim, Ovation, Axcan Pharma, Genentech, and Altus, consulting fees and travel support from Vertex, Axcan Pharma, Boehringer Ingelheim, CSL Bering, Digestive Care, Genentech, GlaxoSmithKline, Gilead, NanoBio, Nektar, Novartis, PTC Therapeutics, Solvay, and Transave, and speaking fees from Roche and Genentech, and serving on the scientific advisory board for Aradigm; Dr. Moss, receiving consulting fees from Vertex, Novartis, Inspire, Genentech, Mpex, PTC Therapeutics, Aridis, AOP/Lantibio, Johnson & Johnson, and 23andMe, grant support from Vertex, Gilead, PTC Therapeutics, Inspire, and GlaxoSmithKline, speaking fees from Novartis, Genentech, and AstraZeneca and travel support from AstraZeneca, Novartis, Vertex, and Genentech, and owning stock in Gilead and Genentech; Dr. Pilewski, receiving grant support from Novartis and Vertex; Dr. Rubenstein, receiving consulting fees from Transave; Dr. Uluer, receiving grant support from Vertex, Boehringer Ingelheim, Education Development Center, and Bayer, speaking fees from Vertex, and travel support from Vertex and Bayer; Dr. Aitken, receiving grant support from Pharmaxis, Transave, Vertex, and PTC Therapeutics and travel support from Pharmaxis and Vertex; Dr. Freedman, receiving grant support from Vertex, Pfizer, Hood Foundation, Gerber Foundation, and Merck; Drs. Dong, Zha, Olson, Ordoñez, and Ms. Stone, being employees of Vertex and holding stock in and receiving travel support from Vertex; and Dr. Ramsey, receiving grants from AlgiPharma, Aradigm, Bayer, CSL Behring, Eurand, GlaxoSmithKline, Inspire, Johnson & Johnson, KaloBios, Mpex, N30 Pharmaceuticals, Novartis, Pharmaxis, PTC Therapeutics, Seed-One Ventures, Transave, and Vertex.
We thank all the subjects involved in the study and the study coordinators, nurses, and technicians performing the nasal potential difference procedure at investigative sites for their contributions, including Churee Pardee and Lisa Monchil (University of Colorado Denver); Heather Young, Ginger Reeves, and Gina Sabbatini (University of Alabama at Birmingham); Lois Brass and Dr. Kelvin MacDonald (Johns Hopkins Medical Institutions); Brooke Noren, Cynthia Williams, and Dale Carlquist (University of Minnesota); Julie Avolio (Hospital for Sick Children, Toronto); Myra Slutsky (St. Michael’s Hospital, Toronto); Cheri Lux, Janice Launspach, and Dr. Mary Teresi (University of Iowa Carver School of Medicine); Roberta Ksenich and Timothy R. Myers (Case Western Reserve University); Carol Barlow, Nadia Bendahmane, and Caroline O’Connor (University of North Carolina at Chapel Hill); Colleen Dunn and Zoe Davies (Stanford University); Elizabeth Hartigan, Sandra Hurban, and Adrienne Horn (University of Pittsburgh); Christina Kubrak (Children’s Hospital of Philadelphia); Erin M. Leone (Children’s Hospital Boston); Alan Genatossio and Sharon McNamara (Seattle Children’s Hospital); and Louis J. Boitano (University of Washington, Seattle). We also thank Dr. Robert J. Beall (Cystic Fibrosis Foundation) and Niki Hoagland (Seattle Children’s Hospital) for contributions and support; Morty Cohen (Seattle Children’s Hospital) for providing solutions for testing the nasal potential difference; John Beamer (Seattle Children’s Hospital) for standard operating procedures related to testing of the nasal potential difference; and Dr. Barry Lubarsky (Vertex Pharmaceuticals) for coordinating the editing and submission of an earlier version of the manuscript. We also thank the following cystic fibrosis treatment centers for referring subjects for this study: University of Utah (Dr. Ted Liou), Yale University (Dr. John McArdle), Children’s Hospital of Michigan (Dr. Debbie Toder), Children’s Hospital Medical Center of Akron (Dr. Nathan Kraynack), and Vanderbilt University (Dr. Bonnie Slovis).