Results 191 to 200 of about 7,856 (243)

Comparison of Reporting Quality in National Cystic Fibrosis Patient Registries: Implications for Identifying Use of Novel CFTR Modulators. [PDF]

Pulm Ther
Tomlinson OW, Mitchelmore P, Williams CA.   +2 more
europepmc   +1 more source

Association between CFTR modulators and changes in iron deficiency markers in cystic fibrosis. [PDF]

J Cyst Fibros
Jia S, Wang Y, Ross MH, Zuckerman JB, Murray S, Han MK, Cahalan SE, Lenhan BE, Best RN, Taylor-Cousar JL, Simon RH, Fitzgerald LJ, Troost JP, Sood SL, Gifford AH.   +14 more
europepmc   +1 more source

Comparison of the Effect of CFTR Modulators <i>elexacaftor</i>/<i>tezacaftor</i>/<i>ivacaftor</i> and <i>lumacaftor</i>/<i>ivacaftor</i> via Serum Human Epididymis Protein 4 Concentration in <i>p.Phe508del-CFTR</i> Homozygous Cystic Fibrosis Patients. [PDF]

J Clin Med
Pócsi M, Fila L, Péterfia C, Halász A, Szanto TG, Mészáros B, Major J, Laki I, Szabó H, Panyi G, Balogh I, Amaral MD, Macek M, Nagy B.   +13 more
europepmc   +1 more source

Erratum: Inflammatory Activity of Epithelial Stem Cell Variants from Cystic Fibrosis Lungs Is Not Resolved by CFTR Modulators. [PDF]

Am J Respir Crit Care Med
europepmc   +1 more source

CFTR Modulators: Current Status and Evolving Knowledge

Seminars in Respiratory and Critical Care Medicine, 2022
AbstractIn the past decade, the medical management of people with cystic fibrosis (pwCF) has changed with the development of small molecules that partially restore the function of the defective CF transmembrane conductance regulator (CFTR) protein and are called CFTR modulators.
Lucile, Regard, Clémence, Martin, Jennifer, Da Silva, Pierre-Régis, Burgel   +3 more
openaire   +3 more sources

CFTR Modulators: Does One Dose Fit All? [PDF]

Journal of Personalized Medicine, 2021
For many people with cystic fibrosis (pwCF), CFTR modulators will be the cornerstone of their treatment. These modulators show robust treatment effects at group level in pwCF with specific mutations. The individual effect however, is variable.
Renske Van Der Meer, Harry Heijerman
exaly   +2 more sources

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Discovery of CFTR modulators for the treatment of cystic fibrosis

Expert Opinion on Drug Discovery, 2021
Cystic fibrosis (CF) is a life-threatening inherited disease caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein, an anion channel expressed at the apical membrane of secretory epithelia. CF leads to multiorgan dysfunction with progressive deterioration of lung function being the major cause of untimely ...
Miquéias Lopes-Pacheco, Nicoletta Pedemonte, Guido Veit   +2 more
openaire   +3 more sources

Revisiting Host-Pathogen Interactions in Cystic Fibrosis Lungs in the Era of CFTR Modulators

International Journal of Molecular Sciences, 2023
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators, a new series of therapeutics that correct and potentiate some classes of mutations of the CFTR, have provided a great therapeutic advantage to people with cystic fibrosis (pwCF).
Carla M P Ribeiro, Matthew G Higgs, Marianne S Muhlebach   +2 more
exaly   +2 more sources

R560S: A class II CFTR mutation that is not rescued by current modulators

Journal of Cystic Fibrosis, 2019
Background: New therapies modulating defective CFTR have started to hit the clinic and others are in trial or under development. The endeavour of drug discovery for CFTR protein rescue is however difficult one since over 2000 mutations have been reported.
Nikhil T Awatade, Sofia S Ramalho, Iris A L Silva   +2 more
exaly   +2 more sources