Literature DB >> 34379998

Elexacaftor/Tezacaftor/Ivacaftor Improved Clinical Outcomes in a Patient with N1303K-CFTR Based on In Vitro Experimental Evidence.

Yunjie Huang1, Grace Paul2, Jesun Lee1, Sunitha Yarlagadda1, Karen McCoy2, Anjaparavanda P Naren1.   

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Year:  2021        PMID： 34379998      PMCID： PMC8759307          DOI： 10.1164/rccm.202101-0090LE

Source DB:  PubMed          Journal:  Am J Respir Crit Care Med        ISSN： 1073-449X            Impact factor:   30.528


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  11 in total

1.  Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Advanced Cystic Fibrosis Lung Disease.

Authors:  Amir Reza Djavid; Alison E Thompson; Alexandria L Irace; Elen Gusman; Kimberly Altman; Emily A DiMango; Claire L Keating
Journal:  Ann Am Thorac Soc       Date:  2021-05-17

2.  Efficacy of elexacaftor/tezacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease.

Authors:  Kate M O'Shea; Orla M O'Carroll; Catherine Carroll; Brenda Grogan; Anna Connolly; Lynda O'Shaughnessy; Trevor T Nicholson; Charles G Gallagher; Edward F McKone
Journal:  Eur Respir J       Date:  2021-02-25       Impact factor: 16.671

3.  Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del.

Authors:  Jennifer L Taylor-Cousar; Anne Munck; Edward F McKone; Cornelis K van der Ent; Alexander Moeller; Christopher Simard; Linda T Wang; Edward P Ingenito; Charlotte McKee; Yimeng Lu; Julie Lekstrom-Himes; J Stuart Elborn
Journal:  N Engl J Med       Date:  2017-11-03       Impact factor: 91.245

4.  Rapid Improvement after Starting Elexacaftor-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and Advanced Pulmonary Disease.

Authors:  Pierre-Régis Burgel; Isabelle Durieu; Raphaël Chiron; Sophie Ramel; Isabelle Danner-Boucher; Anne Prevotat; Dominique Grenet; Christophe Marguet; Martine Reynaud-Gaubert; Julie Macey; Laurent Mely; Annlyse Fanton; Sébastien Quetant; Lydie Lemonnier; Jean-Louis Paillasseur; Jennifer Da Silva; Clémence Martin
Journal:  Am J Respir Crit Care Med       Date:  2021-07-01       Impact factor: 21.405

5.  N1303K and IVS8-5T, clinical presentation within a family with atypical cystic fibrosis.

Authors:  Kim Van Hoorenbeeck; Katrien Storm; Jenneke van den Ende; Martine Biervliet; Kristine N Desager
Journal:  J Cyst Fibros       Date:  2006-11-28       Impact factor: 5.482

6.  Combination potentiator ('co-potentiator') therapy for CF caused by CFTR mutants, including N1303K, that are poorly responsive to single potentiators.

Authors:  Puay-Wah Phuan; Jung-Ho Son; Joseph-Anthony Tan; Clarabella Li; Ilaria Musante; Lorna Zlock; Dennis W Nielson; Walter E Finkbeiner; Mark J Kurth; Luis J Galietta; Peter M Haggie; Alan S Verkman
Journal:  J Cyst Fibros       Date:  2018-06-12       Impact factor: 5.482

7.  Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770.

Authors:  Fredrick Van Goor; Sabine Hadida; Peter D J Grootenhuis; Bill Burton; Dong Cao; Tim Neuberger; Amanda Turnbull; Ashvani Singh; John Joubran; Anna Hazlewood; Jinglan Zhou; Jason McCartney; Vijayalaksmi Arumugam; Caroline Decker; Jennifer Yang; Chris Young; Eric R Olson; Jeffery J Wine; Raymond A Frizzell; Melissa Ashlock; Paul Negulescu
Journal:  Proc Natl Acad Sci U S A       Date:  2009-10-21       Impact factor: 11.205

8.  Rescue of multiple class II CFTR mutations by elexacaftor+tezacaftor+ivacaftor mediated in part by the dual activities of elexacaftor as both corrector and potentiator.

Authors:  Onofrio Laselva; Claire Bartlett; Tarini N A Gunawardena; Hong Ouyang; Paul D W Eckford; Theo J Moraes; Christine E Bear; Tanja Gonska
Journal:  Eur Respir J       Date:  2021-06-17       Impact factor: 16.671

9.  Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination.

Authors:  Guido Veit; Ariel Roldan; Mark A Hancock; Dillon F Da Fonte; Haijin Xu; Maytham Hussein; Saul Frenkiel; Elias Matouk; Tony Velkov; Gergely L Lukacs
Journal:  JCI Insight       Date:  2020-09-17

10.  A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele.

Authors:  Edith T Zemanick; Jennifer L Taylor-Cousar; Jane Davies; Ronald L Gibson; Marcus A Mall; Edward F McKone; Paul McNally; Bonnie W Ramsey; Jonathan H Rayment; Steven M Rowe; Elizabeth Tullis; Neil Ahluwalia; Chenghao Chu; Thang Ho; Samuel M Moskowitz; Sabrina Noel; Simon Tian; David Waltz; Tanya G Weinstock; Fengjuan Xuan; Claire E Wainwright; Susanna A McColley
Journal:  Am J Respir Crit Care Med       Date:  2021-06-15       Impact factor: 21.405
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  3 in total

Review 1.  One Size Does Not Fit All: The Past, Present and Future of Cystic Fibrosis Causal Therapies.

Authors:  Marjolein M Ensinck; Marianne S Carlon
Journal:  Cells       Date:  2022-06-08       Impact factor: 7.666

2.  CFTR Modulator Therapy in an Individual With Cystic Fibrosis Caused by a N1303K CFTR Variant and Infected With Mycobacterium abscessus.

Authors:  E Claire Elson; Paula Capel; Jessica Haynes; Stephanie Duehlmeyer; Michelle Fischer; Hugo Escobar
Journal:  J Pediatr Pharmacol Ther       Date:  2022-05-09

3.  Novel CFTR modulator combinations maximise rescue of G85E and N1303K in rectal organoids.

Authors:  Marjolein M Ensinck; Liesbeth De Keersmaecker; Anabela S Ramalho; Senne Cuyx; Stephanie Van Biervliet; Lieven Dupont; Frauke Christ; Zeger Debyser; François Vermeulen; Marianne S Carlon
Journal:  ERJ Open Res       Date:  2022-04-19
  3 in total

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