Results 61 to 70 of about 10,277 (199)

Optimization of pharmaceutical research and development by early‐phase assessment of investigational medicinal products

British Journal of Clinical Pharmacology, EarlyView.
Small and mid‐sized pharmaceutical innovators often have limited in‐house health economics and market access expertise, and may struggle to align development strategies of investigational medicinal products with health system needs and payer expectations.
Zoltán Kaló, Attila Imre, Gábor Varga, Sándor Kovács, György Tímár, Balázs Nagy   +5 more
wiley   +1 more source

CFTR modulators partially restore the epithelial interferome in Aspergillus infection to improve clinical outcomeResearch in context

EBioMedicine
Summary: Background: The impact of CFTR modulator therapy on host immunity and outcomes in people with Cystic Fibrosis (CF)-related Aspergillus lung disease is poorly defined.
Sarah L. Laverty, Imogen Felton, Michelle Casey, Benjamin Hopwood, Nicholas Simmonds, Darius Armstrong-James, Anand Shah, Peter Kelleher   +7 more
doaj   +1 more source

Molecular basis of cystic fibrosis and modern therapeutic approaches [PDF]

, 2019
Identifikacija molekularne povezanosti između genske nefunkcionalnosti CFTR gena te patofiziološke i kliničke pozadine cistične fibroze omogućila je razvoj suvremenih terapijskih modela koji su primarno utemeljeni na individualiziranom pristupu ...
Dragičević, Dorian
core   +2 more sources

Peptide Nucleic Acids as miRNA Target Protectors for the Treatment of Cystic Fibrosis [PDF]

, 2017
Cystic Fibrosis (CF) is one of the most common life shortening conditions in Caucasians. CF is caused by mutations in the CF Transmembrane Conductance Regulator (CFTR) gene which result in reduced or altered CFTR functionality. Several microRNAs (miRNAs)
Amato, Felice, Borbone, Nicola, Castaldo, Giuseppe, Catalanotti, Bruno, D'Errico, Stefano, Morgillo, CARMINE MARCO, Oliviero, Giorgia, Piccialli, Gennaro, Pinto, Brunella, Santarpia, Giuliano, Zarrilli, Federica   +10 more
core   +1 more source

Long‐term impact of Elexacaftor/Tezacaftor/ivacaftor on pulmonary, nutritional and metabolic outcomes in homozygous F508del cystic fibrosis patients: A real‐world cohort study

British Journal of Clinical Pharmacology, EarlyView.
Aim Elexacaftor/tezacaftor/ivacaftor (ETI) has markedly improved cystic fibrosis (CF) outcomes. However, its long‐term impact on nutrition, metabolism and liver health remains underexplored. We assessed 30‐month changes in pulmonary, nutritional, metabolic and inflammatory markers in people with CF (PwCF) homozygous for F508del.
Nicola Perrotta, Luigi Angelo Fiorito, Roberta Vescovo, Anna Virgilio, Giulia Amato, Giuseppe Cimino   +5 more
wiley   +1 more source

CFTR Modulator Therapy Enhances Peripheral Blood Monocyte Contributions to Immune Responses in People With Cystic Fibrosis

Frontiers in Pharmacology, 2020
BackgroundCFTR modulators decrease some etiologies of CF airway inflammation; however, data indicate that non-resolving airway infection and inflammation persist in individuals with CF and chronic bacterial infections.
Katherine B. Hisert, Katherine B. Hisert, Timothy P. Birkland, Kelly Q. Schoenfelt, Matthew E. Long, Brenda Grogan, Suzanne Carter, W. Conrad Liles, Edward F. McKone, Lev Becker, Anne M. Manicone, Sina A. Gharib   +11 more
doaj   +1 more source

Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression. [PDF]

, 2014
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane regulator (CFTR) that result in reduced anion conductance at the apical membrane of secretory epithelia.
Apaja, Pirjo M., Avramescu, Radu G., Bagdany, Miklos, Borot, Florence, Finkbeiner, Walter E., Hegedus, Tamas, Lukacs, Gergely L., Perdomo, Doranda, Phuan, Puay-Wah, Szollosi, Daniel, Veit, Guido, Verkman, Alan S., Wu, Yu-Sheng   +12 more
core   +1 more source

Targeting neutrophil extracellular traps in metabolic and immune niche: Nanomaterials for diabetes tissue regeneration

BMEMat, EarlyView.
The effects of NETs on regeneration of various diabetic tissues, and strategies targeting NETs for diabetes tissue regeneration. In the diabetic environment, NETs undergo complex metabolic and immune reprogramming, leading to dynamic changes in antibacterial and proinflammatory functions, and affecting regeneration of multiple systemic tissues.
Xinyi Jiang, Muxin Yue, Kai Mao, Boon Chin Heng, Noor Azlin Yahya, Yunsong Liu, Zheng Li   +6 more
wiley   +1 more source

A grumbling concern: A survey of gastrointestinal symptoms in cystic fibrosis in the modulator era [version 2; peer review: 1 approved, 4 approved with reservations]

NIHR Open Research
Background Gastrointestinal symptoms in cystic fibrosis (CF) are common and intrusive to daily life. Relieving gastrointestinal symptoms was identified as an important research priority and previously explored in an international survey in 2018. However,
Vinishaa Premakumar, Vigilius Gleetus, Zoe Elliott, Bu Hayee, Nicola J Rowbotham, Bethinn Evans, Helen Barr, Siobhán B Carr, Daniel Peckham, Alexander Horsley, Alan R Smyth, Natalie Goodchild, Rebecca J Calthorpe   +12 more
doaj   +1 more source

Peripheral Protein Quality Control as a Novel Drug Target for CFTR Stabilizer

Frontiers in Pharmacology, 2018
Conformationally defective cystic fibrosis transmembrane conductance regulator (CFTR) including rescued ΔF508-CFTR is rapidly eliminated from the plasma membrane (PM) even in the presence of a CFTR corrector and potentiator, limiting the therapeutic ...
Ryosuke Fukuda, Tsukasa Okiyoneda
doaj   +1 more source