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Lumacaftor/ivacaftor therapy fails to increase insulin secretion in F508del/F508del CF patients.
Moheet A, Beisang D, Zhang L, Sagel SD, VanDalfsen JM, Heltshe SL, Frederick C, Mann M, Antos N, Billings J, Rowe SM, Moran A; PROSPECT Investigators of the Cystic Fibrosis Foundation Therapeutics Development Network. Moheet A, et al. Among authors: vandalfsen jm. J Cyst Fibros. 2021 Mar;20(2):333-338. doi: 10.1016/j.jcf.2020.09.001. Epub 2020 Sep 8. J Cyst Fibros. 2021. PMID: 32917547 Free PMC article.
Building global development strategies for cf therapeutics during a transitional cftr modulator era.
Mayer-Hamblett N, van Koningsbruggen-Rietschel S, Nichols DP, VanDevanter DR, Davies JC, Lee T, Durmowicz AG, Ratjen F, Konstan MW, Pearson K, Bell SC, Clancy JP, Taylor-Cousar JL, De Boeck K, Donaldson SH, Downey DG, Flume PA, Drevinek P, Goss CH, Fajac I, Magaret AS, Quon BS, Singleton SM, VanDalfsen JM, Retsch-Bogart GZ. Mayer-Hamblett N, et al. Among authors: vandalfsen jm. J Cyst Fibros. 2020 Sep;19(5):677-687. doi: 10.1016/j.jcf.2020.05.011. Epub 2020 Jun 7. J Cyst Fibros. 2020. PMID: 32522463 Free PMC article. Review.
PROMISE: Working with the CF community to understand emerging clinical and research needs for those treated with highly effective CFTR modulator therapy.
Nichols DP, Donaldson SH, Frederick CA, Freedman SD, Gelfond D, Hoffman LR, Kelly A, Narkewicz MR, Pittman JE, Ratjen F, Sagel SD, Rosenfeld M, Schwarzenberg SJ, Singh PK, Solomon GM, Stalvey MS, Kirby S, VanDalfsen JM, Clancy JP, Rowe SM. Nichols DP, et al. Among authors: vandalfsen jm. J Cyst Fibros. 2021 Mar;20(2):205-212. doi: 10.1016/j.jcf.2021.02.003. Epub 2021 Feb 19. J Cyst Fibros. 2021. PMID: 33619012 Free PMC article. Review.
Evaluating the Impact of Stopping Chronic Therapies after Modulator Drug Therapy in Cystic Fibrosis: The SIMPLIFY Clinical Trial Study Design.
Mayer-Hamblett N, Nichols DP, Odem-Davis K, Riekert KA, Sawicki GS, Donaldson SH, Ratjen F, Konstan MW, Simon N, Rosenbluth DB, Retsch-Bogart G, Clancy JP, VanDalfsen JM, Buckingham R, Gifford AH. Mayer-Hamblett N, et al. Among authors: vandalfsen jm. Ann Am Thorac Soc. 2021 Aug;18(8):1397-1405. doi: 10.1513/AnnalsATS.202010-1336SD. Ann Am Thorac Soc. 2021. PMID: 33465316 Free PMC article.