Disparities and therapeutic advances in cystic fibrosis
JB Palla - Pediatric Pulmonology, 2023 - Wiley Online Library
Cystic fibrosis (CF) has seen a multitude of therapeutic advances targeting its downstream
effects. This has led to a steady increase in survival over the past few decades. The recent …
effects. This has led to a steady increase in survival over the past few decades. The recent …
Left behind: the potential impact of CFTR modulators on racial and ethnic disparities in cystic fibrosis
ME McGarry, ER Gibb, GR Oates… - Paediatric respiratory …, 2022 - Elsevier
The advent of CFTR modulators, a genomic specific medication, revolutionized the treatment
of CF for many patients. However, given that these therapeutics were only developed for …
of CF for many patients. However, given that these therapeutics were only developed for …
The future of CFTR modulating therapies for cystic fibrosis
JC Davies - Current Opinion in Pulmonary Medicine, 2015 - journals.lww.com
With the advent of such precision medicine, patient genotype is now highly relevant. Areas
of current unmet need include drugs to cover all mutation classes, increasing access to …
of current unmet need include drugs to cover all mutation classes, increasing access to …
Potential for therapeutic benefit among cystic fibrosis populations excluded from clinical trials or labeling of marketed therapies
DR VanDevanter, SL Heltshe… - Annals of the American …, 2016 - atsjournals.org
Remarkable advances in the management of individuals born with cystic fibrosis (CF) would
not have been realized without empiric trial and error by CF clinicians with treatments …
not have been realized without empiric trial and error by CF clinicians with treatments …
Cystic fibrosis: Highly effective targeted Therapeutics and the impact on sex and racial disparities
K Montemayor, R Jain - Medical Clinics, 2022 - medical.theclinics.com
People with cystic fibrosis (CF) are living longer and healthier lives due to advances in
therapies, aimed at targeting the underlying pathophysiology and more recently at the …
therapies, aimed at targeting the underlying pathophysiology and more recently at the …
Cystic fibrosis patients of minority race and ethnicity less likely eligible for CFTR modulators based on CFTR genotype
ME McGarry, SA McColley - Pediatric pulmonology, 2021 - Wiley Online Library
Background Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are
disease‐modifying medications for cystic fibrosis (CF) and are shown to be efficacious for …
disease‐modifying medications for cystic fibrosis (CF) and are shown to be efficacious for …
Cystic fibrosis: a model system for precision medicine
SL Martiniano, SD Sagel… - Current opinion in …, 2016 - journals.lww.com
CFTR modulators promise to transform the therapeutic landscape in CF in a precision based
fashion. Areas of ongoing research include developing drugs for all mutation classes so that …
fashion. Areas of ongoing research include developing drugs for all mutation classes so that …
The demographics of adverse outcomes in cystic fibrosis
ME McGarry, WA Williams… - Pediatric …, 2019 - Wiley Online Library
Understanding variability in cystic fibrosis (CF) health outcomes requires an understanding
of factors that go far beyond Cystic Fibrosis Transmembrane Receptor (CFTR) function …
of factors that go far beyond Cystic Fibrosis Transmembrane Receptor (CFTR) function …
Searching for a cure for cystic fibrosis. A 25-year quest in a nutshell
B Bosch, K De Boeck - European journal of pediatrics, 2016 - Springer
After 25 years of intensive search, there is not yet a cure for cystic fibrosis (CF). However, the
quest has led to major breakthroughs in understanding the basic disease defect and …
quest has led to major breakthroughs in understanding the basic disease defect and …
Introduction and Epidemiology of Cystic Fibrosis
AG Filbrun, T Lahiri, CL Ren, AG Filbrun… - Handbook of Cystic …, 2016 - Springer
Cystic fibrosis (CF) is an autosomal recessive condition caused by mutations in the gene
encoding the CF transmembrane conductance regulator (CFTR) protein [1]. CFTR plays a …
encoding the CF transmembrane conductance regulator (CFTR) protein [1]. CFTR plays a …
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