Results 101 to 110 of about 975,042 (316)

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 response of S737F and T465N CFTR variants on patient-derived rectal organoids

Orphanet Journal of Rare Diseases
Background Predictions based on patient-derived materials of CFTR modulators efficacy have been performed lately in patient-derived cells, extending FDA-approved drugs for CF patients harboring rare variants.
Karina Kleinfelder, Paola Melotti, Anca Manuela Hristodor, Cristina Fevola, Giovanni Taccetti, Vito Terlizzi, Claudio Sorio   +6 more
doaj   +1 more source

Paediatric development of radiopharmaceutical imaging agents and radioligand therapeutics

British Journal of Clinical Pharmacology, EarlyView.
Abstract This review focuses on the development of radiopharmaceutical imaging agents and radioligand therapeutics for paediatric use. Nuclear medicine plays an important role in the diagnosis and treatment of various childhood conditions, including cancers, infections and brain disorders.
Justin L. Hay, Amandine Manon, Hunter Stephens, S. Y. Amy Cheung, Bart de Keizer, Erik T. te Beek   +5 more
wiley   +1 more source

Lung clearance index short-term variability in cystic fibrosis: a pre-post pulmonary exacerbation study

Italian Journal of Pediatrics
Background Multiple Breath washout (MBW) represents an important tool to detect early a possible pulmonary exacerbation especially in Cystic Fibrosis (CF) disease.
Matteo De Marchis, Enza Montemitro, Alessandra Boni, Alessandra Federici, Daniele Di Giovanni, Luca Cristiani, Renato Cutrera, Alessandro G. Fiocchi   +7 more
doaj   +1 more source

P235 A UK registry study of prophylactic antibiotic use for Staphylococcus aureus in children with cystic fibrosis [PDF]

, 2011
Douglas Lo, M. H. Hurley, Andrew Prayle, Alan R Smyth   +3 more
openalex   +1 more source

P076 Using the Cystic Fibrosis Foundation consensus guidelines to assess the timing of lung transplant referral for individuals with cystic fibrosis in a single large adult UK centre: do they help?

, 2021
Kavita Dave, R. Dunk, S. Madge, Amanda Reed, N.J. Simmonds   +4 more
openalex   +1 more source

Divergent dynamics of inflammatory mediators and multiplex PCRs during airway infection in cystic fibrosis patients and healthy controls: Serial upper airway sampling by nasal lavage [PDF]

, 2022
Nina Erdmann, Theresa Schilling, Julia Hentschel, Thomas Lehmann, Philipp von Bismarck, Tobias Ankermann, Franziska Duckstein, Michael Baier, Carlos Zagoya, Jochen G. Mainz   +9 more
openalex   +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

Review for "Physiological predictors of cardiorespiratory fitness in children and adolescents with cystic fibrosis without ventilatory limitation"

, 2021

openalex   +1 more source

New opportunities for bioscaffold‐enabled spinal cord injury repair

BMEMat, EarlyView.
Schematic illustration of bioscaffolds for spinal cord injury repair. We summarize the effects of bioscaffold properties on SCI repair, highlight different types of bioscaffolds, various fabrication strategies, and in vivo transformations for the clinical development of SCI‐repairing bioscaffolds.
Xiaoqing Qi, Yicheng Fu, Zhaoliang Su, Li Li, Subrata Chakrabarti, Peng Li, Yilun Wu, Fang Liu, Teng Gao, Zhifeng Dong, Lei Liu, Pei Cao   +11 more
wiley   +1 more source