WS23.6 Ivacaftor treatment in patients with cystic fibrosis who have an R117H-CFTR mutation, the KONDUCT study
Moss, R.B, Flume, P.A, Elborn, J.S, Cooke, J, Rowe, S.M, McColley, S.A, Rubenstein, R.C, Higgins, M
Published in Journal of cystic fibrosis (01.06.2014)
Published in Journal of cystic fibrosis (01.06.2014)
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Journal Article
Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
Wainwright, Claire E, Elborn, J. Stuart, Ramsey, Bonnie W, Marigowda, Gautham, Huang, Xiaohong, Cipolli, Marco, Colombo, Carla, Davies, Jane C, De Boeck, Kris, Flume, Patrick A, Konstan, Michael W, McColley, Susanna A, McCoy, Karen, McKone, Edward F, Munck, Anne, Ratjen, Felix, Rowe, Steven M, Waltz, David, Boyle, Michael P
Published in The New England journal of medicine (16.07.2015)
Published in The New England journal of medicine (16.07.2015)
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Journal Article
WS05.04 Safety and efficacy of ivacaftor (IVA) in children aged 1 to <4 months with cystic fibrosis assessed with an innovative clinical trial design
Rosenfeld, M., Singh, A., McColley, S.A., McNally, P., Haseltine, E., Higgins, M., Liu, M., Lu, J., Rodriguez-Romero, V., Shih, J.L., Davies, J.
Published in Journal of cystic fibrosis (01.06.2023)
Published in Journal of cystic fibrosis (01.06.2023)
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P022 Earlier contact with a cystic fibrosis centre is associated with better nutritional outcomes in infants with cystic fibrosis
Martiniano, S.L., Elbert, A., Farrell, P.M., Fisher, N., Ren, C.L., Sontag, M., Wu, R., McColley, S.A.
Published in Journal of cystic fibrosis (01.06.2020)
Published in Journal of cystic fibrosis (01.06.2020)
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Journal Article
P014 Improvement in cystic fibrosis outcomes since newborn screening implementation in the United States
Martiniano, S.L., Elbert, A., Farrell, P.M., Fisher, N., Ren, C.L., Sontag, M., Wu, R., McColley, S.A.
Published in Journal of cystic fibrosis (01.06.2020)
Published in Journal of cystic fibrosis (01.06.2020)
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Journal Article
Inconclusive diagnosis after a positive newborn bloodspot screening result for cystic fibrosis; clarification of the harmonised international definition
Southern, K.W., Barben, J., Gartner, S., Munck, A., Castellani, C., Mayell, S.J., Davies, J.C., Winters, V., Murphy, J., Salinas, D., McColley, S.A., Ren, C.L., Farrell, P.M.
Published in Journal of cystic fibrosis (01.11.2019)
Published in Journal of cystic fibrosis (01.11.2019)
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Journal Article
WS19.2 Long-term safety of lumacaftor/ivacaftor therapy in persons with cystic fibrosis aged 2–5 years homozygous for the F508del-CFTR mutation (F/F)
Hoppe, J., Chilvers, M., Ratjen, F., McNamara, J.J., Owen, C.A., Tian, S., Zahigian, R., Cornell, A.G., McColley, S.A.
Published in Journal of cystic fibrosis (01.06.2020)
Published in Journal of cystic fibrosis (01.06.2020)
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WS01.5 A 2-part, phase 3 single-arm study to evaluate the safety and pharmacokinetics (PK) of lumacaftor/ivacaftor (LUM/IVA) combination therapy in patients (pts) aged 2 to 5 years with cystic fibrosis (CF) homozygous for the F508del-CFTR mutation
Mcnamara, J., Mccolley, S.A., Owen, C.A., Liu, F., Tian, S., Waltz, D., Marigowda, G., Sawicki, G.S.
Published in Journal of cystic fibrosis (01.06.2018)
Published in Journal of cystic fibrosis (01.06.2018)
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Journal Article
WS20-1 An international survey to determine understanding of the designation of infants with an inconclusive diagnosis after newborn bloodspot screening for cystic fibrosis
Murphy, J., Gartner, S., Munck, A., Barben, J., Winters, V., McColley, S.A., Ren, C.L., Farrell, P.M., Southern, K.W.
Published in Journal of cystic fibrosis (01.06.2019)
Published in Journal of cystic fibrosis (01.06.2019)
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Journal Article
Refining the continuum of CFTR-associated disorders in the era of newborn screening
Levy, H., Nugent, M., Schneck, K., Stachiw-Hietpas, D., Laxova, A., Lakser, O., Rock, M., Dahmer, M.K., Biller, J., Nasr, S.Z., Baker, M., McColley, S.A., Simpson, P., Farrell, P.M.
Published in Clinical genetics (01.05.2016)
Published in Clinical genetics (01.05.2016)
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