In patients with cystic fibrosis (CF), clinical trials are of paramount importance. Here, the current status of drug development in CF is discussed and future directions highlighted. Methods for pre-clinical testing of drugs with potential activity in CF patients including relevant animal models are...
We identified 259 trials, with 193 fulfilling the inclusion criteria. Only 63 (33%) of these matched one or more of the JLA priorities showing that current clinical trials poorly reflect the JLA Top 10. By increasing awareness of the Top 10 priorities, it is hoped that this will fuel ...
The European Cystic Fibrosis Society-Clinical Trials Network (ECFS-CTN) has tracked clinic... S Van Koningsbruggen-Rietschel,F Dunlevy,V Bulteel,... - 《Trials》 被引量: 0发表: 2021年 Implementation of microbiota analysis in clinical trials for cystic fibrosis lung infection: Experience from ...
Cystic fibrosis is caused by genetic errors in the CFTR gene that, among other problems, leads to persistent lung infections. Recurring pulmonary infections result in the progressive destruction of normal lung tissue and, eventually, the need for a double lung transplant, often in young, 20- to ...
Between 1999 and 2014 the Cystic Fibrosis Foundation Patient Registry reported an increase in minorities from 5% to 8.2% Latino, from 3 to 4.6% Black and from 1.4 to 3.1% "other". We evaluated the inclusion of minority patients in clinical trials of drugs to treat CF. Objective: To ...
Standardized procedure for measurement of nasal potential difference: an outcome measure in multicenter cystic fibrosis clinical trials. Pediatr Pulmonol 2004; 37: 385–392. Article PubMed Google Scholar Wagener JS, Sontag MK, Sagel SD, Accurso FJ . Update on newborn screening for cystic fibrosis...
fibrosis. Although the mechanisms driving organ-specific fibrosis have not been fully elucidated, drugs targeting these identified aberrant signals have achieved potent anti-fibrotic efficacy in clinical trials. In this review, we briefly introduce the aetiology and epidemiology of several fibrosis diseases...
fibrosis. Although the mechanisms driving organ-specific fibrosis have not been fully elucidated, drugs targeting these identified aberrant signals have achieved potent anti-fibrotic efficacy in clinical trials. In this review, we briefly introduce the aetiology and epidemiology of several fibrosis diseases...
Rationale: Lung clearance index (LCI), measured by multiple breath washout (MBW), is a noninvasive measure of ventilation inhomogeneity that holds promise as an objective physiologic endpoint for clinical trials in infants and preschool children with cystic fibrosis (CF).Objectives: To study the fea...
Cystic fibrosis (CF), a monogenic disease caused by mutations in the CFTR gene on chromosome 7, is complex and greatly variable in clinical expression. Air