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Pediatric

Cystic Fibrosis

Jennifer L. Taylor-Cousar, M.D., Anne Munck, M.D., Edward F. McKone, M.D., Cornelis K. van der Ent, M.D., Ph.D., Alexander Moeller, M.D., Christopher Simard, M.D., Linda T. Wang, M.D., Edward P. Ingenito, M.D., Ph.D., Charlotte McKee, M.D., Yimeng Lu, Ph.D., Julie Lekstrom-Himes, M.D., and J. Stuart Elborn, M.D. "Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del."  The New England Journal of Medicine 2017.

Sosnay PR, Salinas DB, White TB, Ren CL, Farrell PM, Raraigh KS, Girodon E, Castellani C. Applying Cystic Fibrosis Transmembrane Conductance Regulator Genetics and CFTR2 Data to Facilitate Diagnoses. J Pediatr 2017; 181S: S27-S32 e21.

Ren CL, Borowitz DS, Gonska T, Howenstine MS, Levy H, Massie J, Milla C, Munck A, Southern KW. Cystic Fibrosis Transmembrane Conductance Regulator-Related Metabolic Syndrome and Cystic Fibrosis Screen Positive, Inconclusive Diagnosis. J Pediatr 2017; 181S: S45-S51 e41.

Milla CE, Ratjen F, Marigowda G, Liu F, Waltz D, Rosenfeld M. Lumacaftor/Ivacaftor in patients aged 6-11 years with cystic fibrosis and homozygous for F508del-CFTR. American journal of respiratory and critical care medicine 2017; 195: 912-920.

Leung DH, Heltshe SL, Borowitz D, et al. Effects of diagnosis by newborn screening for cystic fibrosis on weight and length in the first year of life. JAMA Pediatrics 2017; 171: 546-554.

Farrell PM, White TB, Ren CL, Hempstead SE, Accurso F, Derichs N, Howenstine M, McColley SA, Rock M, Rosenfeld M, Sermet-Gaudelus I, Southern KW, Marshall BC, Sosnay PR. Diagnosis of Cystic Fibrosis: Consensus Guidelines from the Cystic Fibrosis Foundation. The Journal of Pediatrics 2017; 181: S4-S15.e11.

Floto RA, Olivier KN, Saiman L, Daley CL, Herrmann JL, Nick JA, Noone PG, Bilton D, Corris P, Gibson RL, Hempstead SE, Koetz K, Sabadosa KA, Sermet-Gaudelus I, Smyth AR, van Ingen J, Wallace RJ, Winthrop KL, Marshall BC, Haworth CS. US Cystic Fibrosis Foundation and European Cystic Fibrosis Society consensus recommendations for the management of non-tuberculous mycobacteria in individuals with cystic fibrosis. Thorax 2016; 71 Suppl 1: i1-i22.

Ren CL, Morgan RL, Oermann C, Resnick HE, Brady C, Campbell A, DeNagel R, Guill M, Hoag J, Lipton A, Newton T, Peters S, Willey-Courand DB, Naureckas ET. Cystic Fibrosis Foundation Pulmonary Guidelines. Use of Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy in Patients with Cystic Fibrosis. Annals of the American Thoracic Society 2018; 15: 271-280.

Paranjape SM, Mogayzel PJ Jr. "Cystic fibrosis." Pediatr Rev. 2014 May;35(5):194-205.

Flume PA, Mogayzel PJ Jr, Robinson KA, Rosenblatt RL, Quittell L, Marshall BC; Clinical Practice Guidelines for Pulmonary Therapies Committee; Cystic Fibrosis Foundation Pulmonary Therapies Committee. "Cystic fibrosis pulmonary guidelines: pulmonary complications: hemoptysis and pneumothorax." Am J Respir Crit Care Med. 2010 Aug 1;182(3):298-306.

Stoltz DA, Meyerholz DK, Welsh MJ. Origins of cystic fibrosis lung disease. N Engl J Med. 2015 Jan 22;372(4):351-62. doi: 10.1056/NEJMra1300109. Review. PubMed PMID: 25607428; PubMed Central PMCID: PMC4916857.

Burgener EB, Moss RB. Cystic fibrosis transmembrane conductance regulator modulators: precision medicine in cystic fibrosis. Curr Opin Pediatr. 2018 Jun;30(3):372-377. Review. PubMed PMID: 29538046.