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Results in Journal Journal of Cystic Fibrosis: 10,164

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Peter G Middleton
Published: 1 April 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.03.020

, Gianfranco Alicandro, Mark Oliver, Peter J Lewindon, Grant A Ramm, Chee Y. Ooi, Federico Alghisi, , , Fabiola Corti, et al.
Published: 1 April 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.03.014

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, Chantal Darquenne, Daniël Schuermans, Sylvia Verbanck, Eef Vanderhelst
Published: 1 April 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.03.016

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, Ruth Watson, Wang Yng Lim, Kim Pollard, Christine Etherington, Ian J Clifton, Daniel G Peckham
Published: 1 April 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.03.005

Abstract:
Background Exercise tolerance in people with CF and advanced lung disease is often reduced. While supplemental oxygen can improve oxygenation, it does not affect dyspnoea, fatigue or comfort. Nasal high-flow therapy (NHFT), thanks to its pathophysiological mechanisms, could improve exercise tolerance, saturation and dyspnoea. This study explores the feasibility of conducting a clinical trial of using NHFT in patients with CF during exercise. Methods A pilot, open-label, randomized crossover trial was performed, enroling 23 participants with CF and severe lung disease. Participants completed two treadmill walking test (TWT) with and without NHFT at 24–48 h interval. Primary outcome was trial feasibility, and exploratory outcomes were TWT distance (TWTD), SpO2, transcutaneous CO2, dyspnoea and comfort. Results Recruitment rate was 2.4 subjects/month with 1.3:1 screening-to-randomization ratio. No adverse events caused by NHFT were observed. Tolerability was good and data completion rate was 100%. Twenty subjects (91%) were included in the exploratory study. Mean difference in TWTD on NHFT was 19 m (95% CI [4.8 - 33.1]). SpO2 was similar, but respiratory rate and mean tcCO2 were lower on NHFT (mean difference = -3.9 breaths/min 95% CI [-5.9 - -1.9] and -0.22 kPa 95% CI [-0.4 – 0.04]). NHFT reduced exercise-induced dyspnoea and discomfort. Conclusion Trials using NHFT in patients with CF during exercise are feasible. NHFT appears to improve walking distance, control respiratory rate, CO2, dyspnoea and improve comfort. A larger trial with a longer intervention is feasible and warranted to confirm the impact of NHFT in training programmes for patients with CF.
, R. Casciaro, D. Pirlo, , E. Castagnola, F. Cresta,
Published: 1 April 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.03.024

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Published: 1 April 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.03.010

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Jessica E. Pittman, Umer Khan, Theresa A. Laguna, Sonya Heltshe, Christopher H. Goss,
Published: 1 March 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.02.013

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Laura J. Sherrard, , , , Keyur A. Dave, , Claire E. Wainwright, Keith Grimwood, Hanna E. Sidjabat, David M. Whiley, et al.
Published: 1 March 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.03.007

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Rowena Mills, Riina Rautemaa-Richardson, Stuart Wilkinson, , Anirban Maitra,
Published: 1 March 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.03.012

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, Shanal Kumar, Chris Daley, Shrinkhala Dawadi, , Erin Carr, Georgia Soldatos
Published: 1 March 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.03.004

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, Nicole Mayer-Hamblett, Christopher H. Goss, George Z. Retsch-Bogart, Jill M. VanDalfsen, Patricia Burks, Daniel Rosenbluth, John Paul Clancy, Amy Hoffman, David P. Nichols
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 195-197; doi:10.1016/j.jcf.2020.12.007

Abstract:
The Severe Acute Respiratory Syndrome Coronavirus (SARS-CoV-2) global pandemic significantly impacted CF clinical research within the Cystic Fibrosis Foundation Therapeutics Development Network (CFF TDN). A Research Electronic Data Capture (REDCap) survey was developed and sent to network sites to monitor and understand the impact on research teams, ongoing and anticipated clinical research, and specific clinical and research procedures. Key findings indicated an early impact on participant enrollment, research team stability, and procedures such as spirometry and sputum induction. These trends steadily improved over the months as research activities began to recover across the TDN. While SARS-CoV-2 created a significant challenge it also highlights new opportunities to expand CF research with greater focus on data collection outside of research centers and increased access for remote participation.
, , Maria Gabriela Tupayachi Ortiz, Daniel Dorgan, Raksha Jain, Elizabeth A. Poth, Robert C. Fifer, Yun Jin M. Kim, Angela G. Shoup, Patrick A. Flume
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 288-294; doi:10.1016/j.jcf.2020.11.020

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Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 188-190; doi:10.1016/j.jcf.2020.12.017

Marc A. Sala, Michael Alexander, Basil Khuder, , , , Jing Liu, , Paul A. Reyfman
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 356-363; doi:10.1016/j.jcf.2020.12.018

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, A. Baines, A. Paynter, S.L. Heltshe, J. VanDalfsen, M. Jain, S.M. Rowe, S.D. Sagel
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 213-219; doi:10.1016/j.jcf.2020.11.008

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, John T. Brinton, Tim Vigers, Melanie Cree-Green, Philip S. Zeitler, Kristen J. Nadeau,
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 339-345; doi:10.1016/j.jcf.2020.08.020

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Isaac T Bernhardt, Philip Moore, Sarah Currie,
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 330-332; doi:10.1016/j.jcf.2020.08.018

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, , Jay B. Hilliard, Michael W. Konstan
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 257-263; doi:10.1016/j.jcf.2020.05.008

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, Xiayi Ma, Jenna Sykes, Sanja Stanojevic, Adele Coriati, Anne L. Stephenson
Published: 1 March 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.03.008

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Rende Gu, Ryan J Longenecker, Jennifer Homan,
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 271-277; doi:10.1016/j.jcf.2020.02.014

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, Sivagurunathan Sutharsan, , Ross C. Klingsberg, Rainald Fischer, Steven M. Rowe, Paul K. Audhya, Neil Ahluwalia, Xiaojun You, Thomas J. Ferro, et al.
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 228-233; doi:10.1016/j.jcf.2020.06.001

Abstract:
Increased rates of respiratory adverse events have been observed in people ≥12 years of age with cystic fibrosis homozygous for the Phe508del-CFTR mutation treated with lumacaftor/ivacaftor, particularly in those with percent predicted forced expiratory volume in 1 s (ppFEV1) of <40%. We evaluated the safety, tolerability, and efficacy of tezacaftor/ivacaftor in people with cystic fibrosis homozygous for Phe508del-CFTR who discontinued lumacaftor/ivacaftor due to treatment-related respiratory signs or symptoms. Participants ≥12 years of age with cystic fibrosis homozygous for Phe508del-CFTR with ppFEV1 of ≥25% and ≤90% were randomized 1:1 and treated with tezacaftor/ivacaftor or placebo for 56 days. Of 97 participants, 94 (96.9%) completed the study. The primary endpoint was incidence of predefined respiratory adverse events of special interest (chest discomfort, dyspnea, respiration abnormal, asthma, bronchial hyperreactivity, bronchospasm, and wheezing): tezacaftor/ivacaftor, 14.0%; placebo, 21.3%. The adverse events were mild or moderate in severity. None were serious or led to treatment interruption or discontinuation. Overall, the discontinuation rate was similar between groups. The mean (SD) ppFEV1 at baseline was 44.6% (16.1%) with tezacaftor/ivacaftor and 48.0% (18.1%) with placebo. The posterior mean difference in absolute change in ppFEV1 from baseline to the average value of days 28 and 56 was 2.7 percentage points with tezacaftor/ivacaftor vs placebo. Tezacaftor/ivacaftor was generally safe, well tolerated, and efficacious in people ≥12 years of age with cystic fibrosis homozygous for Phe508del-CFTR with ppFEV1 of ≥25% and ≤90% who previously discontinued lumacaftor/ivacaftor due to treatment-related respiratory signs or symptoms.
, Elena Amelina, Cori L. Daines, Brett Charlton, Joanna Leadbetter, Alessandro Guasconi, Moira L. Aitken
Published: 1 March 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.02.011

Abstract:
Background : Mannitol is a mucoactive hyperosmotic agent used as add-on therapy in patients with cystic fibrosis (CF), administered twice-daily (BID) via a small, portable, breath-actuated dry-powder inhaler. This study was conducted to provide confirmatory evidence of mannitol's efficacy and safety in adults. Methods : This multicenter, double-blind, randomized, parallel-group, controlled clinical trial recruited adults (aged ≥18 years) with CF, and forced expiratory volume in 1 second (FEV1) 40–90% predicted. Subjects received either mannitol 400 mg or mannitol 50 mg (control), BID via dry-powder inhaler for 26 weeks. Primary endpoint: FEV1 averaged over the 26-week treatment period. Results : Of 423 subjects randomized (209 or 214 receiving mannitol 400 mg BID or control, respectively), 373 (88.2%) completed the study, with a similar proportion completing in the two groups. For FEV1 averaged over 26 weeks, mannitol 400 mg BID was statistically superior to control (adjusted mean difference 54 mL [95% CI 8, 100 mL]; p = 0.020). This was supported by sensitivity analyses of the primary endpoint, and by observed improvements in secondary pulmonary function endpoints (eg, absolute adjusted mean difference in percent predicted FEV1 averaged over 26 weeks 1.21% [0.07%, 2.36%]; p = 0.037). Adverse events were mainly mild or moderate in severity, with treatment-related adverse events in 15.5 and 12.2% of subjects receiving mannitol 400 mg BID and control, respectively. Conclusions : In adults with CF, mannitol 400 mg BID inhaled as a dry-powder statistically significantly improved lung function (FEV1) compared with control, with this improvement supported by sensitivity analyses and secondary pulmonary function endpoints. Mannitol had a good overall safety and tolerability profile. ClinicalTrials.gov: NCT02134353.
Natalie Franz, Hannah Rapp, Ryan N. Hansen, Laura S. Gold, Christopher H. Goss, Noah Lechtzin,
Published: 1 March 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.02.014

The publisher has not yet granted permission to display this abstract.
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 193-194; doi:10.1016/j.jcf.2021.03.001

Michael M. Rey,
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 191-192; doi:10.1016/j.jcf.2021.02.008

, Malcolm Gleser, Alexis Johns, Erik Larsen, Jay Vachhani
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 278-283; doi:10.1016/j.jcf.2020.07.001

, V Carnovale, , S Quattrucci, D Taruscio, G Floridia, A Amato, Gianluca Ferrari, Giuseppe Campagna, Marco Salvatore
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 372-373; doi:10.1016/j.jcf.2021.01.008

The publisher has not yet granted permission to display this abstract.
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 234-242; doi:10.1016/j.jcf.2020.11.003

Abstract:
Background Tezacaftor (TEZ)/ivacaftor (IVA) is an approved CFTR modulator shown to be efficacious and generally safe and well tolerated in people ≥12 years of age with cystic fibrosis (CF) homozygous for the F508del-CFTR mutation or heterozygous for the F508del-CFTR mutation and a residual function mutation. Although previous studies with IVA alone showed clinical benefits in people with CFTR gating mutations, TEZ/IVA has not yet been evaluated in a Phase 3 study of participants heterozygous for F508del-CFTR and a gating mutation (F/gating genotypes). Here, we present results from a randomized, double-blind, IVA-controlled, parallel-group, Phase 3 study assessing the efficacy, safety, and pharmacokinetics (PK) of TEZ/IVA in participants ≥12 years of age with F/gating genotypes. Methods Enrolled participants entered a 4-week IVA run-in period to create a stable IVA baseline. Participants were then randomized to receive IVA or TEZ/IVA for 8 weeks in an active comparator treatment period (ACTP). The primary endpoint was absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1). Key secondary endpoints were relative change in ppFEV1 and absolute change in CF Questionnaire–Revised respiratory domain score. Secondary endpoints included absolute change in sweat chloride (SwCl) concentration, PK parameters, and safety. All endpoints except PK parameters and safety were assessed from baseline through Week 8. Results Sixty-nine participants (92.0%) in the IVA group and 75 participants (98.7%) in the TEZ/IVA group completed treatment. No improvements were seen in efficacy endpoints from baseline at the end of the IVA run-in period through the end of the ACTP in the IVA group. No significant differences in ppFEV1 or any key secondary endpoint were observed between the IVA and TEZ/IVA groups. SwCl concentrations decreased more in the TEZ/IVA versus IVA group during the ACTP. The safety profile and PK parameters of TEZ/IVA were consistent with those of previous studies in participants ≥12 years of age with CF. Conclusions This Phase 3 study showed that the dual-combination regimen of TEZ/IVA demonstrated clinical efficacy but did not have significantly greater clinical efficacy than IVA alone in participants ≥12 years of age with F/gating genotypes. However, as reported in other studies, TEZ/IVA was generally safe and well tolerated (NCT02412111).
, Maurizio Bonati, Rita Campi, Rita Pescini, Carlo Castellani
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 198-204; doi:10.1016/j.jcf.2020.12.016

The publisher has not yet granted permission to display this abstract.
, Imogen Felton, Phillip James, , Diana Bilton, Silke Schelenz, Michael R. Loebinger, , Nicholas J. Simmonds, Miriam F. Moffatt
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 295-302; doi:10.1016/j.jcf.2020.05.013

Abstract:
The prevalence of fungal disease in cystic fibrosis (CF) and non-CF bronchiectasis is increasing and the clinical spectrum is widening. Poor sensitivity and a lack of standard diagnostic criteria renders interpretation of culture results challenging. In order to develop effective management strategies, a more accurate and comprehensive understanding of the airways fungal microbiome is required. The study aimed to use DNA sequences from sputum to assess the load and diversity of fungi in adults with CF and non-CF bronchiectasis. Next generation sequencing of the ITS2 region was used to examine fungal community composition (n = 176) by disease and underlying clinical subgroups including allergic bronchopulmonary aspergillosis, chronic necrotizing pulmonary aspergillosis, non-tuberculous mycobacteria, and fungal bronchitis. Patients with no known active fungal disease were included as disease controls. ITS2 sequencing greatly increased the detection of fungi from sputum. In patients with CF fungal diversity was lower, while burden was higher than those with non-CF bronchiectasis. The most common operational taxonomic unit (OTU) in patients with CF was Candida parapsilosis (20.4%), whereas in non-CF bronchiectasis sputum Candida albicans (21.8%) was most common. CF patients with overt fungal bronchitis were dominated by Aspergillus spp., Exophiala spp., Candida parapsilosis or Scedosporium spp. This study provides a framework to more accurately characterize the extended spectrum of fungal airways diseases in adult suppurative lung diseases.
Shamsah Kazani, David J. Rowlands, , Julie Milojevic, Jose Alcantara, Ieuan Jones, Kenneth Kulmatycki, Surendra Machineni, Lidia Mostovy, Ian Nicholls, et al.
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 250-256; doi:10.1016/j.jcf.2020.11.002

Abstract:
Background This is the first-in-human study of icenticaftor, an oral potentiator of the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) channel. Restoration of CFTR activity has shown significant clinical benefits, but more studies are needed to address all CFTR mutations. Methods Safety, pharmacodynamics/pharmacokinetics of icenticaftor were evaluated in a randomized, double-blind, placebo-controlled study in healthy volunteers. Efficacy was assessed in adult CF patients with ≥1 pre-specified CFTR Class III or IV mutation (150 and 450 mg bid), or homozygous for F508del mutation (450 mg bid). Primary efficacy endpoint was change from baseline in lung clearance index (LCI2.5). Secondary endpoints included %predicted FEV1 and sweat chloride level. Results Class IV mutations were present in 22 patients, Class III in 2 (both S549N), and 25 were homozygous for F508del. Icenticaftor was well-tolerated in healthy and CF subjects with no unexpected events or discontinuations in the CF groups. The most frequent study-drug related adverse events in CF patients were nausea (12.2%), headache (10.2%), and fatigue (6.1%). Icenticaftor 450 mg bid for 14 days showed significant improvements in all endpoints versus placebo in patients with Class III and IV mutations; mean %predicted FEV1 increased by 6.46%, LCI2.5 decreased by 1.13 points and sweat chloride decreased by 8.36 mmol/L. No significant efficacy was observed in patients homozygous for a single F508del. Conclusions Icenticaftor was safe and well-tolerated in healthy volunteers and CF patients, and demonstrated clinically meaningful changes in lung function and sweat chloride level in CF patients with Class III and IV CFTR mutations. ClinicalTrials.gov: NCT02190604
, Alexandra Coronel Palacios, Aravinda Thiagalingam, Peter G Middleton
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20; doi:10.1016/j.jcf.2020.07.016

The publisher has not yet granted permission to display this abstract.
, Andrew Lee, Noreen Caine, Susan C Charman, Diana Bilton
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 324-329; doi:10.1016/j.jcf.2020.08.001

, Malcolm Brodlie, Christopher O'Brien, Matthew F. Thomas
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20; doi:10.1016/j.jcf.2020.10.004

Abstract:
We report the first case of biodegradable airway stent insertion for a patient with bronchomalacia and cystic fibrosis (CF). This female infant with antenatally diagnosed cystic fibrosis developed respiratory distress by three weeks of age. On lower airway examination she was found to have severe left main stem bronchomalacia causing left upper lobe hyperinflation and subsequent right upper lobe collapse. By eight weeks of age she developed life-threatening respiratory failure requiring high pressure invasive ventilation. A biodegradable bronchial stent was inserted to the left main bronchus at thirteen weeks of age with successful extubation from invasive respiratory support four days later. A second biodegradable stent was inserted nine weeks later, due to persistent bronchomalacia following stent degradation and redevelopment of oxygen requirement. She was discharged home off all respiratory support eight weeks later and has remained well, requiring no further surgical intervention for bronchomalacia to date, now age three years.
Alice C. Eastman, Rhonda G. Pace, , Melis Atalar Aksit, Briana Vecchio-Pagán, Anh-Thu N. Lam, Wanda K. O'Neal, Scott M. Blackman, ,
Published: 1 March 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.02.007

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, Katarina Vukovojac, Jenna Sykes, , Elizabeth Tullis, Anne L. Stephenson
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 243-249; doi:10.1016/j.jcf.2020.07.017

The publisher has not yet granted permission to display this abstract.
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20; doi:10.1016/s1569-1993(21)00062-x

, Sonya L. Heltshe, Michelle Skalland, Noah Lechtzin, Dave Nichols, Christopher H. Goss
Published: 1 March 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.02.012

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Davis Jaclyn, Nesmith Andrew, Perkins Ryan, Bailey Julianna, Siracusa Christopher, , M. Powers, Sawicki Gregory S,
Published: 1 March 2021
Journal of Cystic Fibrosis; doi:10.1016/j.jcf.2021.03.009

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, Scott H. Donaldson, Carla A. Frederick, Steven D. Freedman, Daniel Gelfond, , Andrea Kelly, Michael R. Narkewicz, Jessica E. Pittman, Felix Ratjen, et al.
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 205-212; doi:10.1016/j.jcf.2021.02.003

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, Helga Mikkelsen, Lucy McCahon, Samantha Grogan, William Ditcham, David W. Reid, Iain Lamont, Stephen M. Stick, Barry Clements
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 316-323; doi:10.1016/j.jcf.2020.12.004

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Thaís P. Mello, Michaela Lackner, Marta H. Branquinha,
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 303-309; doi:10.1016/j.jcf.2020.12.001

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Nikhil T. Awatade, Sharon L. Wong, , Elvis Pandzic, Iveta Slapetova, Ling Zhong, Nihan Turgutoglu, Laura K. Fawcett, Renee M. Whan, , et al.
Published: 1 March 2021
Journal of Cystic Fibrosis, Volume 20, pp 364-371; doi:10.1016/j.jcf.2020.12.019

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