Results 11 to 20 of about 1,531 (166)

Physiologically Based Pharmacokinetic Modeling of Elexacaftor/Tezacaftor/Ivacaftor in Infants With Cystic Fibrosis. [PDF]

CPT Pharmacometrics Syst Pharmacol
ABSTRACT Ivacaftor is the only cystic fibrosis transmembrane conductance regulator modulator approved for infants ≥ 1 month. The elexacaftor/tezacaftor/ivacaftor combination, approved for children aged ≥ 2 years, has been shown to significantly slow CF progression.
Truong NH, Benaboud S, Bouazza N, Deneuville E, Mornand A, Reix P, Ribault M, Burgel PR, Froelicher-Bournaud L, Rouillon S, Sanoufi MR, Lui G, Urien S, Tréluyer JM, Sermet-Gaudelus I, Foissac F, Modul‐CF study group.   +16 more
europepmc   +2 more sources

Beyond the Lung. Impact of Elexacaftor/Tezacaftor/Ivacaftor on Sinonasal Disease in Children With Cystic Fibrosis. [PDF]

Int Forum Allergy Rhinol
ABSTRACT Background Elexacaftor/tezacaftor/ivacaftor (ETI) is a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) therapy that improves pulmonary function and chronic rhinosinusitis (CRS) in cystic fibrosis (CF) adults with at least one copy of the F508del CFTR mutation.
Petitjean M, Letierce A, Bonnel AS, Reix P, Deneuville E, Stremler N, Luscan R, Couloigner V, Mely L, Bessaci K, Labbe G, Marguet C, Kelly-Aubert M, Hassani F, Sermet-Gaudelus I, Simon F, MODUL‐CF Study Group.   +16 more
europepmc   +2 more sources

Newborn Screening for Cystic Fibrosis Is Associated With the Lowest Healthcare Costs: A 10-Year Observational Follow-Up Study in France. [PDF]

Pediatr Pulmonol
ABSTRACT Objectives This study aims to study the healthcare (HC) costs associated with cystic fibrosis (CF) in children diagnosed prenatally (ANT), through newborn screening (NBS), after birth due to meconium ileus (MI), or later based on symptoms (LS).
Guyot E, Deygas F, Belhassen M, Berard M, Van Ganse E, Sermet-Gaudelus I, Tiaiba S, Dubus JC, Durieu I, Reix P.   +9 more
europepmc   +2 more sources

The Value of Enhancing Sonographic Phenotyping to Improve the Diagnostic Yield of Noninvasive Prenatal Diagnosis (NIPD) for Achondroplasia. [PDF]

Prenat Diagn
ABSTRACT Objectives Achondroplasia is the most common form of skeletal dysplasia and is usually suspected in the third trimester of pregnancy based on abnormal sonographic findings. Non‐invasive prenatal diagnosis (NIPD), based on the detection of pathogenic FGFR3 variants in maternal plasma, provides an accurate genetic confirmation.
Verebi C, Gravrand V, Guerini C, Anselem O, Vaucouleur N, Boimard LO, Ben Ouazzou R, Demirtas C, Leturcq F, Bienvenu T, Tsatsaris V, Pannier E, Nectoux J.   +12 more
europepmc   +2 more sources

3D Automated Segmentation of Bronchial Abnormalities on Ultrashort Echo Time MRI: A Quantitative MR Outcome in Cystic Fibrosis. [PDF]

J Magn Reson Imaging
ABSTRACT Background Cystic fibrosis (CF) monitoring relies on computed tomography (CT), but ultra‐short echo time MRI (UTE‐MRI) offers a radiation‐free alternative. However, its clinical adoption is hindered by the laborious and subjective manual analysis, which prevents standardized quantification of bronchial abnormalities.
Bouzid AIH, Benlala I, de Senneville BD, Baldacci F, Macey J, Benhassen W, Su P, Bui S, Zysman M, Bustin A, Berger P, Dournes G.   +11 more
europepmc   +2 more sources

Rationally Designed InhA Inhibitors: A Comparative Anti-Tubercular Activity Study of Sulfonate Esters of Isoniazid Hydrazones and Their Structurally Flexible Benzyl Analogues. [PDF]

Chem Biol Drug Des
A comparative anti‐tubercular evaluation of sulfonate esters of isoniazid hydrazones and their structurally flexible benzyl analogues disclosed several potent compounds against Mycobacterium tuberculosis with the representative compound displaying an MIC of 0.078 μg/mL.
Kadima MG, Mishra S, Kumar G, Seboletswe P, Roquet-Banères F, Foubert M, Kremer L, Karpoormath R, Singh P.   +8 more
europepmc   +2 more sources

Beneficial short-term effect of autogenic drainage on peripheral resistance in childhood cystic fibrosis disease

BMC Pulmonary Medicine, 2022
Background Airway clearance techniques are supposed to be a necessary adjunct for the enhancement of impaired peripheral clearance in cystic fibrosis (CF).
Plamen Bokov, Michèle Gerardin, Géraldine Brialix, Emmanuelle Da Costa Noble, Romain Juif, Antonia Vital Foucher, Laurence Le Clainche, Véronique Houdouin, Benjamin Mauroy, Christophe Delclaux   +9 more
doaj   +1 more source

Corrigendum: Long term NIV in an infant with Hallermann-Streiff syndrome: a case report and overview of respiratory morbidity [PDF]

Frontiers in Pediatrics
S. Guerin, S. Blanchon, Q. de Halleux, V. Bayon, T. Ferry   +4 more
doaj   +2 more sources

Estudo da freqüência de diabetes mellitus e intolerância à glicose em pacientes com fibrose cística Study of the frequency of diabetes mellitus and glucose intolerance in patients with cystic fibrosis

Jornal de Pediatria, 2001
OBJETIVO: avaliar a freqüência de diabetes mellitus (DM) e de intolerância à glicose nos pacientes com fibrose cística seguidos no Serviço de Gastropediatria do HC-FMRP-USP.
Flávia A.A. de Castro, Maria I.M. Fernandes, Renan M. Montenegro Júnior, Milton C. Foss   +3 more
doaj   +1 more source

Correction to: In vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis: a pilot study

BMC Medicine, 2018
The original article [1] contains errors in Table 1 affecting some of the presented oligonucleotide sequences and readthrough values in Table 1.
I. Sermet-Gaudelus, M. Renouil, A. Fajac, L. Bidou, B. Parbaille, S. Pierrot, N. Davy, E. Bismuth, P. Reinert, G. Lenoir, J. F. Lesure, J. P. Rousset, A. Edelman   +12 more
doaj   +1 more source