| Year |
Citation |
Score |
| 2023 |
Wainwright C, McColley SA, McNally P, Powers M, Ratjen F, Rayment JH, Retsch-Bogart G, Roesch E, Ahluwalia N, Chin A, Chu C, Lu M, Menon P, Waltz D, Weinstock T, et al. Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged ≥6 Years with Cystic Fibrosis and At Least One Allele: A Phase 3, Open-Label Clinical Trial. American Journal of Respiratory and Critical Care Medicine. PMID 37154609 DOI: 10.1164/rccm.202301-0021OC |
0.32 |
|
| 2023 |
Uluer AZ, MacGregor G, Azevedo P, Indihar V, Keating C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Rubenstein RC, Taylor-Cousar JL, Tullis E, Yonker LM, Chu C, Lam AP, ... ... Waltz D, et al. Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials. The Lancet. Respiratory Medicine. PMID 36842446 DOI: 10.1016/S2213-2600(22)00504-5 |
0.403 |
|
| 2021 |
Sutharsan S, McKone EF, Downey DG, Duckers J, MacGregor G, Tullis E, Van Braeckel E, Wainwright CE, Watson D, Ahluwalia N, Bruinsma BG, Harris C, Lam AP, Lou Y, Moskowitz SM, ... ... Waltz D, et al. Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial. The Lancet. Respiratory Medicine. PMID 34942085 DOI: 10.1016/S2213-2600(21)00454-9 |
0.457 |
|
| 2021 |
Barry PJ, Mall MA, Álvarez A, Colombo C, de Winter-de Groot KM, Fajac I, McBennett KA, McKone EF, Ramsey BW, Sutharsan S, Taylor-Cousar JL, Tullis E, Ahluwalia N, Jun LS, Moskowitz SM, ... ... Waltz D, et al. Triple Therapy for Cystic Fibrosis -Gating and -Residual Function Genotypes. The New England Journal of Medicine. 385: 815-825. PMID 34437784 DOI: 10.1056/NEJMoa2100665 |
0.37 |
|
| 2020 |
McCoy KS, Heijerman H, Taylor-Cousar JL, Waltz D, Sosnay PR, Ramsey BW, Rowe S, Welter J. Transparency and diversity in cystic fibrosis research - Authors' reply. Lancet (London, England). 396: 602. PMID 32861305 DOI: 10.1016/S0140-6736(20)30897-7 |
0.346 |
|
| 2019 |
Middleton PG, Mall MA, Dřevínek P, Lands LC, McKone EF, Polineni D, Ramsey BW, Taylor-Cousar JL, Tullis E, Vermeulen F, Marigowda G, McKee CM, Moskowitz SM, Nair N, Savage J, ... ... Waltz D, et al. Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele. The New England Journal of Medicine. 381: 1809-1819. PMID 31697873 DOI: 10.1056/Nejmoa1908639 |
0.466 |
|
| 2019 |
Heijerman HGM, McKone EF, Downey DG, Van Braeckel E, Rowe SM, Tullis E, Mall MA, Welter JJ, Ramsey BW, McKee CM, Marigowda G, Moskowitz SM, Waltz D, Sosnay PR, Simard C, et al. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial. Lancet (London, England). PMID 31679946 DOI: 10.1016/S0140-6736(19)32597-8 |
0.481 |
|
| 2019 |
Taylor-Cousar JL, Mall MA, Ramsey BW, McKone EF, Tullis E, Marigowda G, McKee CM, Waltz D, Moskowitz SM, Savage J, Xuan F, Rowe SM. Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two alleles. Erj Open Research. 5. PMID 31218221 DOI: 10.1183/23120541.00082-2019 |
0.515 |
|
| 2019 |
McNamara JJ, McColley SA, Marigowda G, Liu F, Tian S, Owen CA, Stiles D, Li C, Waltz D, Wang LT, Sawicki GS. Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2-5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study. The Lancet. Respiratory Medicine. PMID 30686767 DOI: 10.1016/S2213-2600(18)30460-0 |
0.444 |
|
| 2018 |
Davies JC, Moskowitz SM, Brown C, Horsley A, Mall MA, McKone EF, Plant BJ, Prais D, Ramsey BW, Taylor-Cousar JL, Tullis E, Uluer A, McKee CM, Robertson S, Shilling RA, ... ... Waltz D, et al. VX-659-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles. The New England Journal of Medicine. PMID 30334693 DOI: 10.1056/Nejmoa1807119 |
0.519 |
|
| 2018 |
Keating D, Marigowda G, Burr L, Daines C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Sass LA, Tullis E, McKee CM, Moskowitz SM, Robertson S, Savage J, Simard C, ... ... Waltz D, et al. VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles. The New England Journal of Medicine. PMID 30334692 DOI: 10.1056/Nejmoa1807120 |
0.509 |
|
| 2018 |
McColley SA, Konstan MW, Ramsey BW, Stuart Elborn J, Boyle MP, Wainwright CE, Waltz D, Vera-Llonch M, Marigowda G, Jiang JG, Rubin JL. Lumacaftor/Ivacaftor reduces pulmonary exacerbations in patients irrespective of initial changes in FEV. Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society. PMID 30146268 DOI: 10.1016/J.Jcf.2018.07.011 |
0.442 |
|
| 2018 |
Bevivino A, Coiana A, Fogazzi A, Timelli F, Signorini S, Lucarelli M, Morelli P, Padoan R, Giordani B, Amato A, Majo F, Ferrari G, Quattrucci S, Minicucci L, Floridia G, ... ... Waltz D, et al. Abstracts from the 23rd Italian congress of Cystic Fibrosis and the 13th National congress of Cystic Fibrosis Italian Society Italian Journal of Pediatrics. 44. DOI: 10.1186/S13052-017-0430-4 |
0.409 |
|
| 2018 |
Davies J, Colombo C, Tullis E, Mckee C, Desouza C, Waltz D, Savage J, Fisher M, Shilling R, Moskowitz S, Robertson S, Tian S, Taylor-Cousar J, Rowe S. WS01.6 Preliminary safety and efficacy of triple combination CFTR modulator regimens in cystic fibrosis Journal of Cystic Fibrosis. 17: S3. DOI: 10.1016/S1569-1993(18)30124-3 |
0.443 |
|
| 2018 |
Mcnamara J, Mccolley S, Owen C, Liu F, Tian S, Waltz D, Marigowda G, Sawicki G. WS01.5 A 2-part, phase 3 single-arm study to evaluate the safety and pharmacokinetics (PK) of lumacaftor/ivacaftor (LUM/IVA) combination therapy in patients (pts) aged 2 to 5 years with cystic fibrosis (CF) homozygous for the F508del-CFTR mutation Journal of Cystic Fibrosis. 17: S2-S3. DOI: 10.1016/S1569-1993(18)30123-1 |
0.52 |
|
| 2017 |
Taylor-Cousar JL, Jain M, Barto TL, Haddad T, Atkinson J, Tian S, Tang R, Marigowda G, Waltz D, Pilewski J. Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR. Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society. PMID 29126871 DOI: 10.1016/J.Jcf.2017.09.012 |
0.502 |
|
| 2017 |
Ratjen F, Hug C, Marigowda G, Tian S, Huang X, Stanojevic S, Milla CE, Robinson PD, Waltz D, Davies JC. Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial. The Lancet. Respiratory Medicine. PMID 28606620 DOI: 10.1016/S2213-2600(17)30215-1 |
0.523 |
|
| 2017 |
Brody A, Nagle S, Hug C, Marigowda G, Waltz D, Goldin J, Ratjen F, Wang L. S93 Effect of lumacaftor/ivacaftor on total, bronchiectasis, and air trapping computed tomography (ct) scores in children homozygous for f508del-cftr: exploratory imaging substudy Thorax. 72. DOI: 10.1136/Thoraxjnl-2017-210983.99 |
0.4 |
|
| 2017 |
Nagle S, Brody A, Woods J, Johnson K, Wang L, Marigowda G, Waltz D, Goldin J, Ratjen F, Hug C. P254 Feasibility of ultrashort echo time (ute) mri to evaluate the effect of lumacaftor/ivacaftor therapy in children with cystic fibrosis (cf) homozygous for f508del Thorax. 72. DOI: 10.1136/Thoraxjnl-2017-210983.396 |
0.362 |
|
| 2017 |
Taylor-Cousar J, Jain M, Barto T, Haddad T, Atkinson J, Tian S, Tang R, Marigowda G, Waltz D, Pilewski J. 55 Lumacaftor/ivacaftor (LUM/IVA) in patients (pts) with cystic fibrosis (CF) and advanced lung disease homozygous for F508del-CFTR: a 24-week open-label study Journal of Cystic Fibrosis. 16: S78. DOI: 10.1016/S1569-1993(17)30420-4 |
0.493 |
|
| 2017 |
Ratjen F, Tian S, Marigowda G, Hug C, Huang X, Stanojevic S, Milla C, Robinson P, Waltz D, Davies J. WS13.4 Efficacy and safety of lumacaftor/ivacaftor (LUM/IVA) in patients (pts) aged 6–11 years (yrs) with cystic fibrosis (CF) homozygous for F508del-CFTR: a randomized placebo (PBO)-controlled phase 3 trial Journal of Cystic Fibrosis. 16: S24. DOI: 10.1016/S1569-1993(17)30234-5 |
0.49 |
|
| 2016 |
Konstan MW, McKone EF, Moss RB, Marigowda G, Tian S, Waltz D, Huang X, Lubarsky B, Rubin J, Millar SJ, Pasta DJ, Mayer-Hamblett N, Goss CH, Morgan W, Sawicki GS. 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. The Lancet. Respiratory Medicine. PMID 28011037 DOI: 10.1016/S2213-2600(16)30427-1 |
0.452 |
|
| 2016 |
Rowe SM, McColley SA, Rietschel E, Li X, Bell SC, Konstan MW, Marigowda G, Waltz D, Boyle MP. Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR. Annals of the American Thoracic Society. PMID 27898234 DOI: 10.1513/Annalsats.201609-689Oc |
0.519 |
|
| 2016 |
Milla CE, Ratjen F, Marigowda G, Liu F, Waltz D, Rosenfeld M. Lumacaftor/Ivacaftor in Patients Aged 6-11 Years With Cystic Fibrosis Homozygous for F508del-CFTR. American Journal of Respiratory and Critical Care Medicine. PMID 27805836 DOI: 10.1164/Rccm.201608-1754Oc |
0.497 |
|
| 2016 |
Elborn JS, Ramsey BW, Boyle MP, Konstan MW, Huang X, Marigowda G, Waltz D, Wainwright CE. Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup: a pooled analysis. The Lancet. Respiratory Medicine. PMID 27298017 DOI: 10.1016/S2213-2600(16)30121-7 |
0.484 |
|
| 2016 |
Sullivan J, Accurso F, Marigowda G, Beusmans J, Geho D, Zhang E, Moss R, Waltz D. WS04.2 Improvement in inflammatory biomarkers in patients (pts) with cystic fibrosis (CF) homozygous for the F508del-CFTR mutation treated with lumacaftor (LUM) and ivacaftor (IVA) Journal of Cystic Fibrosis. 15: S6. DOI: 10.1016/S1569-1993(16)30079-0 |
0.488 |
|
| 2015 |
Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, Colombo C, Davies JC, De Boeck K, Flume PA, Konstan MW, McColley SA, McCoy K, McKone EF, Munck A, ... ... Waltz D, et al. Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. The New England Journal of Medicine. 373: 220-31. PMID 25981758 DOI: 10.1056/Nejmoa1409547 |
0.427 |
|
| 2015 |
Seliger V, Bai Y, Volkova N, Tian S, Waltz D. 196 Prevalence of cataracts in a population of cystic fibrosis patients homozygous for the F508del mutation Journal of Cystic Fibrosis. 14: S108. DOI: 10.1016/S1569-1993(15)30373-8 |
0.422 |
|
| 2015 |
Elborn J, Ramsey B, Boyle M, Wainwright C, Konstan M, Huang X, Marigowda G, Waltz D. 143 Lumacaftor/ivacaftor combination therapy in CF patients homozygous for F508del-CFTR with severe lung dysfunction Journal of Cystic Fibrosis. 14: S94. DOI: 10.1016/S1569-1993(15)30320-9 |
0.38 |
|
| 2015 |
Elborn J, Ramsey B, Boyle M, Wainwright C, Konstan M, Huang X, Marigowda G, Waltz D. WS01.3 Lumacaftor in combination with ivacaftor in patients with cystic fibrosis who are homozygous for the F508del-CFTR mutation Journal of Cystic Fibrosis. 14: S1. DOI: 10.1016/S1569-1993(15)30003-5 |
0.406 |
|
| 2014 |
Tiddens HA, Puderbach M, Venegas JG, Ratjen F, Donaldson SH, Davis SD, Rowe SM, Sagel SD, Higgins M, Waltz DA. Novel outcome measures for clinical trials in cystic fibrosis. Pediatric Pulmonology. PMID 25641878 DOI: 10.1002/Ppul.23146 |
0.325 |
|
| 2014 |
Boyle MP, Bell SC, Konstan MW, McColley SA, Rowe SM, Rietschel E, Huang X, Waltz D, Patel NR, Rodman D. A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. The Lancet. Respiratory Medicine. 2: 527-38. PMID 24973281 DOI: 10.1016/S2213-2600(14)70132-8 |
0.46 |
|
| 2013 |
Uluer AZ, Waltz DA, Kalish LA, Adams S, Gerard C, Ericson DA. Inhaled amiloride and tobramycin solutions fail to eradicate Burkholderia dolosa in patients with cystic fibrosis. Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society. 12: 54-9. PMID 23046902 DOI: 10.1016/J.Jcf.2012.06.006 |
0.371 |
|
| 2013 |
Boyle M, Bell S, Konstan M, Colley SM, Flume P, Kang L, Wu Y, Waltz D, Patel N. WS7.4 Lumacaftor, an investigational CFTR corrector, in combination with ivacaftor, a CFTR potentiator, in CF patients with the F508del-CFTR mutation: Phase 2 interim analysis Journal of Cystic Fibrosis. 12: S14. DOI: 10.1016/S1569-1993(13)60043-0 |
0.494 |
|
| 2007 |
Moss RB, Milla C, Colombo J, Accurso F, Zeitlin PL, Clancy JP, Spencer LT, Pilewski J, Waltz DA, Dorkin HL, Ferkol T, Pian M, Ramsey B, Carter BJ, Martin DB, et al. Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial. Human Gene Therapy. 18: 726-32. PMID 17685853 DOI: 10.1089/Hum.2007.022 |
0.379 |
|
| 2007 |
Dovey M, Aitken ML, Emerson J, McNamara S, Waltz DA, Gibson RL. Oral corticosteroid therapy in cystic fibrosis patients hospitalized for pulmonary exacerbation: a pilot study. Chest. 132: 1212-8. PMID 17646219 DOI: 10.1378/Chest.07-0843 |
0.397 |
|
| 2004 |
Moss RB, Rodman D, Spencer LT, Aitken ML, Zeitlin PL, Waltz D, Milla C, Brody AS, Clancy JP, Ramsey B, Hamblett N, Heald AE. Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial. Chest. 125: 509-21. PMID 14769732 DOI: 10.1378/Chest.125.2.509 |
0.323 |
|
| 2003 |
Gibson RL, Emerson J, McNamara S, Burns JL, Rosenfeld M, Yunker A, Hamblett N, Accurso F, Dovey M, Hiatt P, Konstan MW, Moss R, Retsch-Bogart G, Wagener J, Waltz D, et al. Significant microbiological effect of inhaled tobramycin in young children with cystic fibrosis. American Journal of Respiratory and Critical Care Medicine. 167: 841-9. PMID 12480612 DOI: 10.1164/Rccm.200208-855Oc |
0.362 |
|
| 2001 |
Aitken ML, Moss RB, Waltz DA, Dovey ME, Tonelli MR, McNamara SC, Gibson RL, Ramsey BW, Carter BJ, Reynolds TC. A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease. Human Gene Therapy. 12: 1907-16. PMID 11589832 DOI: 10.1089/104303401753153956 |
0.302 |
|
| 2000 |
Robinson W, Waltz DA. FEV(1) as a guide to lung transplant referral in young patients with cystic fibrosis. Pediatric Pulmonology. 30: 198-202. PMID 10973037 DOI: 10.1002/1099-0496(200009)30:3<198::AID-PPUL3>3.0.CO;2-E |
0.307 |
|
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