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PROMISE: Working with the CF community to understand emerging clinical and research needs for those treated with highly effective CFTR modulator therapy.
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.
Multiple observational research studies are now being conducted to better understand the impacts of this important therapeutic milestone on long-term outcomes, patient care needs, and future research priorities. ...
Multiple observational research studies are now being conducted to better understand the impacts of this important therapeutic milestone on …
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.
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.
The protocol is composed of two concurrent randomized controlled trials designed to evaluate the independent short-term effects of discontinuing hypertonic saline or dornase alfa, enabling individuals on both therapies to participate in one or both trials. ...
The protocol is composed of two concurrent randomized controlled trials designed to evaluate the independent short-term effects of di …
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