Results 41 to 50 of about 4,770 (180)

Effect of CFTR Modulators on Respiratory Function in Adults with Cystic Fibrosis [PDF]

open access: yes, 2021
Cystic Fibrosis (CF) is an autosomal recessive disorder that shortens one’s life due to its effect on the cystic fibrosis transmembrane regulator (CFTR) gene.
Severson, Melissa
core   +2 more sources

The era of CFTR modulators: improvements made and remaining challenges [PDF]

open access: yes, 2020
The entry into the clinic of CFTR modulators such as TRIKAFTA has significantly improved life for ∼90% CF patients carrying one or two F508del mutations but challenges remain for rare CFTR mutations and the management of lung infections @SaraOcana1 https:
Avolio, Julie   +3 more
core   +2 more sources

Individualized approach to elexacaftor/tezacaftor/ivacaftor dosing in cystic fibrosis, in response to self-reported anxiety and neurocognitive adverse events: A case series

open access: yesFrontiers in Pharmacology, 2023
The prevalence of mental health disorders is high among people with Cystic Fibrosis. The psychological symptoms in CF are associated with poor adherence, worse treatment outcomes, and greater health utilization/cost.
Hisham Ibrahim   +19 more
doaj   +1 more source

Lingering Identity as Chronically Ill and the Unanticipated Effects of Life-Changing Precision Medicine in Cystic Fibrosis: A Case Report

open access: yesJournal of Patient Experience, 2021
Cystic fibrosis (CF) is the leading genetic disease among Caucasians; however, advances in diagnosis and treatment have improved both quality and quantity of life for those affected.
Sigrid Ladores PhD, RN, PNP, CNE, FAAN   +1 more
doaj   +1 more source

Malassezia Folliculitis following Triple Therapy for Cystic Fibrosis

open access: yesMedicina, 2022
Triple-combination therapy with elexacaftor, tezacaftor and ivacaftor has been recently approved for cystic fibrosis patients with at least one F508del mutation in the transmembrane conductance regulator of the cystic fibrosis gene.
Federica Li Pomi   +8 more
doaj   +1 more source

Overview of CFTR Modulators and Gene Therapy [PDF]

open access: yes, 2020
Individuals with cystic fibrosis (CF) have seen a substantial change in their life expectancy since the introduction of coordinated multi-disciplinary care.
Kotsimbos, Tom   +2 more
core   +1 more source

Impact of CFTR Modulators on Beta-Cell Function in Children and Young Adults with Cystic Fibrosis [PDF]

open access: yes, 2022
Background: To date, no consistent data are available on the possible impact of CFTR modulators on glucose metabolism. The aim of this study was to test the hypothesis that treatment with CFTR modulators is associated with an improvement in the key ...
Bonadonna R. C.   +14 more
core   +1 more source

Uncovering Cystic Fibrosis Carrier: Insights From a Heterozygous CFTR‐F508del Rabbit Model

open access: yesInternational Forum of Allergy &Rhinology, EarlyView.
ABSTRACT Background Chronic rhinosinusitis (CRS) is a heterogeneous inflammatory disorder frequently associated with impaired mucociliary clearance and bacterial infection. Individuals carrying a single cystic fibrosis transmembrane conductance regulator (CFTR) mutation exhibit partial CFTR dysfunction and are increasingly recognized as being at risk ...
Do‐Yeon Cho   +9 more
wiley   +1 more source

Impact of CFTR modulator use on outcomes in people with severe cystic fibrosis lung disease

open access: yesEuropean Respiratory Review, 2020
Drug compounds that augment the production and activity of the cystic fibrosis (CF) transmembrane regulator (CFTR) have revolutionised CF care. Many adults and some children with CF suffer advanced and severe lung disease or await lung transplantation ...
Michal Shteinberg   +1 more
doaj   +1 more source

Aplasia Cutis Congenita in the Setting of Maternal Cystic Fibrosis

open access: yesAnnals of Internal Medicine: Clinical Cases, 2022
Aplasia cutis congenita is a rare scalp defect. An affected neonate was delivered after in utero exposure beginning in the midtrimester to elexacaftor/tezacaftor/ivacaftor for maternal cystic fibrosis management. Although aplasia cutis congenita has many
Govind Kallumkal, Robert Egerman
doaj   +1 more source

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