Expanding access to CFTR modulators for rare mutations: The utility of n-of-1 trials
J Cyst Fibros
.
2020 Jan;19(1):1-2.
doi: 10.1016/j.jcf.2019.11.011.
Epub 2019 Dec 10.
Authors
Amalia S Magaret
1
,
Nicole Mayer-Hamblett
2
,
Donald VanDevanter
3
Affiliations
1
Departments of Pediatrics and Biostatistics, University of Washington, Seattle, WA, USA. Electronic address: amag@uw.edu.
2
Departments of Pediatrics and Biostatistics, University of Washington, Seattle, WA, USA.
3
Department of Pediatrics, Case Western Reserve University School of Medicine, Cleveland, OH, USA.
PMID:
31831338
DOI:
10.1016/j.jcf.2019.11.011
No abstract available
Publication types
Editorial
Comment
MeSH terms
Aminophenols
Cystic Fibrosis Transmembrane Conductance Regulator / genetics
Cystic Fibrosis*
Humans
Mutation
Quinolones
Substances
Aminophenols
Quinolones
Cystic Fibrosis Transmembrane Conductance Regulator
ivacaftor