Clinical trials
SPEEDING UP ACCESS TO NEW DRUGS FOR CYSTIC FIBROSIS: CONSIDERATIONS FOR CLINICAL TRIAL DESIGN AND DELIVERY
Authors: On behalf of the European CF Society (ECFS) Strategic Planning Task Force on ‘Speeding up access to new drugs for CF’: Margarida Amaral6, Kris de Boeck7, Jane C Davies1, […]
THERANOSTICS BY TESTING CFTR MODULATORS IN PATIENT-DERIVED MATERIALS: THE CURRENT STATUS AND A PROPOSAL FOR SUBJECTS WITH RARE CFTR MUTATIONS
Authors: Margarida Amaral1, Kris De Boeck2 On behalf of the ECFS Strategic Planning Task Force on ‘Speeding up access to new drugs for CF’: Margarida Amaral1, Kris De Boeck2, Jane […]
ALIGNING PATIENTS’ NEEDS TOWARDS A COMPREHENSIVE CF RESEARCH PROGRAM
Authors: Jacquelien J. Noordhoek a, Vincent A.M. Gulmans a, Harry G.M. Heijerman b, Cornelis K. van der Ent b Affiliations: a Dutch Cystic Fibrosis Foundation (NCFS) b University Medical Center […]
A SPECIALIZED METHOD OF SPUTUM COLLECTION AND PROCESSING FOR THERAPEUTIC INTERVENTIONS IN CYSTIC FIBROSIS
Authors: McElvaney OJ, Gunaratnam C, Reeves EP, McElvaney NG. Affiliations: Irish Centre for Genetic Lung Disease, Royal College of Surgeons in Ireland, Beaumont Hospital, Dublin, Ireland. What was your […]
CORRESPONDENCE BETWEEN SYMPTOMS AND PREFERENCE-BASED HEALTH STATUS MEASURES IN THE STOP STUDY
Authors: Laura S. Golda, Donald L. Patrickb, Ryan N. Hansenc, Valeria Beckettd, Christopher H. Gosse and Larry Kesslerb Affiliations: aDepartment of Radiology, University of Washington, Seattle, Washington, United States bDepartment […]
APPLICATION OF MULTIPLE EVENT ANALYSIS AS AN ALTERNATIVE APPROACH TO STUDYING PULMONARY EXACERBATIONS AS AN OUTCOME MEASURE
Authors: Elizabeth Juarez-Colunga1,2, Rosenfeld Margaret3, Edith T. Zemanick4, Brandie Wagner1,4 Affiliations: 1Department of Biostatistics and Informatics, University of Colorado School of Public Health, 2Adult and Child Consortium for Health Outcomes […]
DO PATIENTS WITH CYSTIC FIBROSIS PARTICIPATING IN CLINICAL TRIALS DEMONSTRATE PLACEBO RESPONSE? A META-ANALYSIS
Authors: Julie Cotona,b, Ha-Hai Lea, Victor Veuilleta, Perrine Janiaudc, Michel Cucheratd, Behrouz Kassai-Koupaia,e, François Gueyffiera,e, Philippe Reixa,b. Affiliations: aUMR 5558 CNRS, Equipe EMET. Université Claude Bernard Lyon 1, Lyon France […]
ASSESSMENT OF SAFETY AND EFFICACY OF LONG-TERM TREATMENT WITH COMBINATION LUMACAFTOR AND IVACAFTOR THERAPY IN PATIENTS WITH CYSTIC FIBROSIS HOMOZYGOUS FOR THE F508DEL-CFTR MUTATION (PROGRESS): A PHASE 3, EXTENSION STUDY
Citation: Konstan MW, McKone EF, Moss RB, et al. Lancet Respir Med. 2017 Feb;5(2):107-118. What was your research question? What is the long-term safety profile and treatment effectiveness of […]
SPX-101 IS STABLE IN AND RETAINS FUNCTION AFTER EXPOSURE TO CYSTIC FIBROSIS SPUTUM
Authors: Juliana I. Sesma, Bryant Wu, Timothy J. Stuhlmiller, David W. Scott Affiliations: Spyryx Biosciences, Durham, NC 27713 What was your research question? We wanted to understand the stability […]
COMPARISON OF LUNG CLEARANCE INDEX DETERMINED BY WASHOUT OF N2 AND SF6 IN INFANTS AND PRESCHOOL CHILDREN WITH CYSTIC FIBROSIS
Authors: Mirjam Stahla,b,c, Cornelia Joachima,b,c, Mark O. Wielpützc,d, and Marcus A. Malla,b,c,e,f Affiliations: aDivision of Pediatric Pulmonology and Allergy and Cystic Fibrosis Center, Department of Pediatrics, University of Heidelberg, Im […]
ELUFORSEN: A POTENTIAL NEW TREATMENT OPTION FOR PEOPLE WITH CYSTIC FIBROSIS (CF) DUE TO THE F508DEL GENE MUTATION?
Authors: Isabelle Sermet-Gaudelusa, John P. Clancyb, David P. Nicholsc, Jerry A. Nickd, Kris De Boecke, George M. Solomonf, Marcus A. Mallg,h,i, James Bolognesej, Florilene Bouissetk, Wilhelmina den Hollanderl, Nicolas Paquette-Lamontagnel,m, […]