CFTR modulators

DISEASE PROGRESSION IN PATIENTS WITH CYSTIC FIBROSIS TREATED WITH IVACAFTOR: DATA FROM NATIONAL US AND UK REGISTRIES

Authors: Nataliya Volkovaa, Kristin Moya, Jennifer Evansb, Daniel Campbella, Simon Tiana, Christopher Simarda, Mark Higginsc, Michael W. Konstand, Gregory S. Sawickie, Alexander Elbertf, Susan C. Charmang, Bruce C. Marshallf, Diana […]

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MEASURED FETAL AND NEONATAL EXPOSURE TO LUMACAFTOR AND IVACAFTOR DURING PREGNANCY AND WHILE BREASTFEEDING

Authors: Aaron T Trimble1, Cameron McKinzie2, Mary Terrell3, Elizabeth Stringer4, Charles Esther Jr3 Affiliations: 1Department of Medicine, Division of Pulmonary & Critical Care Medicine, 2Eshelman School of Pharmacy, 3Department of […]

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CONTINUOUS GLUCOSE MONITORING IN YOUTH WITH CYSTIC FIBROSIS TREATED WITH LUMACAFTOR-IVACAFTOR

Authors: Angel Li1, Tim Vigers2, Laura Pyle3, Edith Zemanick4, Kristen Nadeau2, Scott D. Sagel4, Christine L. Chan2 Affiliations: 1University of Colorado School of Medicine, University of Colorado Anschutz Medical Campus, […]

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R560S: A CLASS II CFTR MUTATION THAT IS NOT RESCUED BY CURRENT MODULATORS

Authors: Nikhil T Awatade1, Sofia Ramalho1, Iris AL Silva1, Verónica Felício1, Hugo M Botelho1, Eyleen de Poel2, Annelotte Vonk2, Jeffrey M Beekman2, Carlos M Farinha1, Margarida D Amaral1 Affiliations: 1University […]

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RATE AND PREDICTORS OF PRESCRIPTION OF LUMACAFTOR-IVACAFTOR IN THE 18 MONTHS FOLLOWING APPROVAL IN THE UNITED STATES

Authors: Gregory S. Sawicki1, Aliza K. Fink2, Michael S. Schechter3, Deena R. Loeffler2, Nicole Mayer-Hamblett4  Affiliations: 1Division of Respiratory Diseases, Boston Children’s Hospital, Boston, Massachusetts 2Cystic Fibrosis Foundation, Bethesda MD […]

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LUMACAFTOR/IVACAFTOR REDUCES PULMONARY EXACERBATIONS IN PATIENTS IRRESPECTIVE OF INITIAL CHANGES IN FEV1

Authors: Susanna A. McColley,a Michael W. Konstan,b Bonnie W. Ramsey,c J. Stuart Elborn,d Michael P. Boyle,e Claire E. Wainwright,f David Waltz,g Montserrat Vera-Llonch,g Gautham Marigowda,g John G. Jiang,g Jaime L. […]

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HUMAN EPIDIDYMIS PROTEIN 4 (HE4) LEVELS INVERSELY CORRELATE WITH LUNG FUNCTION IMPROVEMENT (DELTA FEV1) IN CYSTIC FIBROSIS PATIENTS RECEIVING IVACAFTOR TREATMENT

Authors: Béla Nagy Jr1, Zsolt Bene2, Zsolt Fejes1, Sonya L. Heltshe3, David Reid4, Nicola J. Ronan5, 3, Yvonne McCarthy5, Daniel Smith4, Attila Nagy6, Elizabeth Joseloff7, György Balla2,8, János Kappelmayer1, Milan […]

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ITALIAN AND NORTH AMERICAN DIETARY INTAKE AFTER IVACAFTOR TREATMENT FOR CYSTIC FIBROSIS GATING MUTATIONS

Authors: Nina Sainath a, Joan Schall a, Chiara Bertolaso b, Carolyn McAnlis a, Virginia A. Stallings a,c Affiliations: aDivision of Gastroenterology, Hepatology and Nutrition, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, […]

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LUMACAFTOR-RESCUED F508DEL-CFTR HAS A MODIFIED BICARBONATE PERMEABILITY

Authors: Loretta Ferrera1, Debora Baroni2 and Oscar Moran2 Affiliations: 1Istituto Giannina Gaslini, U.O.C. Genetica Medica, Genova, Italy 2CNR, Istituto di Biofisica, Genova, Italy   What was your research question? The […]

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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 […]

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