Journal of Cystic Fibrosis最新文献

{"title":"Cardiometabolic risk factors in adults with cystic fibrosis undergoing elexacaftor/tezacaftor/ivacaftor therapy","authors":"Andrea Gramegna ,&nbsp;Massimiliano Ruscica ,&nbsp;Gloria Leonardi ,&nbsp;Chiara Macchi ,&nbsp;Isabella Fichtner ,&nbsp;Gianmarco Putti ,&nbsp;Margherita Carnevale Schianca ,&nbsp;Leonardo Terranova ,&nbsp;Gianfranco Alicandro ,&nbsp;Francesco Blasi","doi":"10.1016/j.jcf.2024.11.009","DOIUrl":"10.1016/j.jcf.2024.11.009","url":null,"abstract":"<div><div>The introduction of elexacaftor/tezacaftor/ivacaftor (ETI) therapy has further extended life expectancy of adults with cystic fibrosis (awCF), highlighting the need for increased attention to potential long-term health issues. Given the increasing prevalence of cardiovascular diseases in the ageing population and the presence of cardiovascular risk factors associated with CF, understanding the impact of ETI on cardiometabolic risk factors is a crucial clinical concern. The aim of our prospective observational study was to explore early changes in cardiac and metabolic biomarkers after 6 months of ETI therapy. A total of 58 consecutive awCF were enrolled during clinical stability at the Adult CF Center of the Policlinico Hospital in Milan, Italy between January 2021 and June 2022. Blood samples were obtained before ETI initiation and after 6 months, and underwent central processing for an extended panel of cardiometabolic biomarkers. We observed a rise in cholesterol, triglycerides, apolipoprotein-B and adipokine levels, while inflammatory markers decreased. The direct relationship between leptin and adiponectin suggest a disruption in the normal regulatory mechanisms that control these hormones, potentially leading to metabolic imbalances, such as increased risk of obesity and cardiovascular events. The impact of ETI on cardiovascular risk in awCF is heterogeneous and while it improves some risk factors, such as chronic inflammation, it has a worsening effect on lipoproteins. Our findings suggest that the dysregulation of adipokines could be a potential cause of the metabolic disturbances observed in awCF.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 53-56"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791736","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Suicidal behaviour and CFTR modulators: A case series and WHO database disproportionality analysis","authors":"Inès Nidegger ,&nbsp;Julie Macey ,&nbsp;Marine Ferey ,&nbsp;Allison Singier ,&nbsp;Marie Tournier ,&nbsp;Justine Perino ,&nbsp;Francesco Salvo","doi":"10.1016/j.jcf.2024.09.020","DOIUrl":"10.1016/j.jcf.2024.09.020","url":null,"abstract":"<div><h3>Background</h3><div>A highly effective therapy involving elexacaftor, tezacaftor, and ivacaftor (ETI) for cystic fibrosis (CF) patients has recently raised safety concerns regarding potential psychiatric disorders. The manuscript reports cases of suicide attempts in patients receiving ETI and investigates putative causality using the WHO spontaneous reporting database.</div></div><div><h3>Methods</h3><div>First, four cases of suicide attempts/self-injury are described. Second, a disproportionality analysis was conducted using spontaneous reports collected in Vigibase through the standardised MedDRA Query (narrow version) \"Suicide/Self-injury\" and ETI exposure. Reporting Odds Ratio (ROR) was calculated for the main and subgroup (i/suicide attempt, ii/suicidal ideation) analyses. Sensitivity analyses were performed with variations in exposure, to ivacaftor/lumacaftor to assess the intrinsic psychiatric risk of CF patients, and paracetamol as a positive control for suicide attempt and a negative one for suicidal ideation. Exposure to reduced-dose ETI was studied to evaluate the dose-gradient effect.</div></div><div><h3>Results</h3><div>Four cases of suicide attempt/self-injury occurred 3 to 13 months after ETI initiation in CF patients and were reported to the Bordeaux Pharmacovigilance centre. Aside, in Vigibase, ETI is associated with an increased likelihood of reporting suicidal behaviour (ROR 2.5, 95 % CI[2.1; 2.8]). A signal of disproportionate reporting was found for the subgroup of suicide attempts (1.4, 95 % CI[1.2; 1.8]), unlike ivacaftor/lumacaftor, which was associated only with the risk of reporting suicidal ideation. Significant ROR values were also found for reduced-dose ETI for all psychiatric effects studied except suicide attempt.</div></div><div><h3>Conclusions</h3><div>ETI exposure is related with increased reporting of suicidal behaviour. A potential dose-dependent effect merits further investigation.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 33-39"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142466568","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"ETD001: A novel inhaled ENaC blocker with an extended duration of action in vivo","authors":"Henry Danahay ,&nbsp;Clive McCarthy ,&nbsp;Thomas Schofield ,&nbsp;Roy Fox ,&nbsp;Holly Charlton ,&nbsp;Sarah Lilley ,&nbsp;Juan Sabater ,&nbsp;Matthias Salathe ,&nbsp;Nathalie Baumlin ,&nbsp;Stephen P Collingwood ,&nbsp;Martin Gosling","doi":"10.1016/j.jcf.2024.06.002","DOIUrl":"10.1016/j.jcf.2024.06.002","url":null,"abstract":"<div><h3>Background</h3><div>Inhibiting ENaC in the airways of people with cystic fibrosis (pwCF) is hypothesized to enhance mucociliary clearance (MCC) and provide clinical benefit. Historically, inhaled ENaC blockers have failed to show benefit in pwCF challenging this hypothesis. It is however unknown whether the clinical doses were sufficient to provide the required long duration of action in the lungs and questions whether a novel candidate could offer advantages where others have failed?</div></div><div><h3>Methods</h3><div>Dose-responses with the failed ENaC blockers (VX-371, BI 1265162, AZD5634, QBW276) together with ETD001 (a novel long acting inhaled ENaC blocker) were established in a sheep model of MCC and were used to predict clinically relevant doses that would provide a long-lasting enhancement of MCC in pwCF. In each case, dose predictions were compared with the selected clinical dose.</div></div><div><h3>Results</h3><div>Each of the failed candidates enhanced MCC in the sheep model. Translating these dose-response data to human equivalent doses, predicted that substantially larger doses of each candidate, than were evaluated in clinical studies, would likely have been required to achieve a prolonged enhancement of MCC in pwCF. In contrast, ETD001 displayed a long duration of action (≥16 h) at a dose level that was well tolerated in Phase 1 clinical studies.</div></div><div><h3>Conclusions</h3><div>These data support that the ENaC blocker hypothesis is yet to be appropriately tested in pwCF. ETD001 has a profile that enables dosing at a level sufficient to provide a long duration of action in a Phase 2 clinical study in pwCF scheduled for 2024.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 72-78"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141293424","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"CF Ferrets exposed to in utero ivacaftor do not develop lens abnormalities","authors":"Jennifer L. Taylor-Cousar ,&nbsp;Shahab Fakhari ,&nbsp;Lacina Allison ,&nbsp;Doug J. Bartels ,&nbsp;Raksha Jain ,&nbsp;Sushan Han","doi":"10.1016/j.jcf.2024.09.007","DOIUrl":"10.1016/j.jcf.2024.09.007","url":null,"abstract":"","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 19-20"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142288298","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Changes in factors associated with inhaled antibiotic prescriptions for people with cystic fibrosis over time in the U.S.","authors":"Marianne S. Muhlebach ,&nbsp;Jane She ,&nbsp;Eric Y. Zhang ,&nbsp;Jonathan D. Cogen ,&nbsp;Michael R. Kosorok","doi":"10.1016/j.jcf.2024.09.017","DOIUrl":"10.1016/j.jcf.2024.09.017","url":null,"abstract":"<div><h3>Rationale</h3><div>CF care guidelines recommend chronic inhaled antibiotics for chronic <em>Pseudomonas aeruginosa</em> (Pa) lung infection. These medications are costly, time consuming and prescription needs may change with improved outcomes.</div></div><div><h3>Objectives</h3><div>We determined the proportion of pwCF with chronic, intermittent or negative Pa infection categories, their clinical and demographic characteristics, factors associated with inhaled antibiotic prescription and changes between 2011 and 2019.</div></div><div><h3>Methods</h3><div>This cohort study using the U.S. CF Foundation patient registry for pwCF &gt;2 years, no prior lung transplant, and with ≥3 respiratory cultures/year determined chronic inhaled antibiotics (≥3 months per calendar year) and Pa infection status from encounter level data. Outcomes and odds of prescription for relevant clinical factors were evaluated using generalized estimating equation models with additional interaction between the predictor and the calendar year to examine changes of predictors over time.</div></div><div><h3>Results</h3><div>Proportion of pwCF with chronic and intermittent Pa decreased and antibiotic prescription rates increased for these groups and decreased for Pa negative pwCF. Hispanic ethnicity, female sex, pancreatic insufficiency, CF diabetes, and ivacaftor/lumacaftor were associated with higher antibiotic prescriptions for each Pa status. Among Pa-negative pwCF prescriptions were higher with <em>Burkholderia spp</em>. (1.17, (CI₉₅ 1.03,1.34)) or MRSA (OR 1.45, (1.26,1.68)) but decreased between 2011 and 2019. For <em>Aspergillus</em> OR increased to 1.6,(1.3,1.8) in 2019. Prescriptions for pwCF on ivacaftor decreased, becoming lower in 2019 for chronic (OR 0.7, (0.5,0.8)) and Pa-negative pwCF (OR 0.7, (0.5,0.8)).</div></div><div><h3>Conclusions</h3><div>Factors predicting inhaled antibiotic prescription differed between 2011 and 2019 indicating changes in health and care for pwCF even prior to triple-modulators.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 98-104"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142400430","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Glucagon-like-peptide-1 agonist therapy in adults with cystic fibrosis","authors":"Sanghoon Park ,&nbsp;Raksha Jain ,&nbsp;Sasan Mirfakhraee","doi":"10.1016/j.jcf.2024.08.005","DOIUrl":"10.1016/j.jcf.2024.08.005","url":null,"abstract":"<div><div>Glucagon-like-peptide-1 (GLP-1) agonists are commonly used to improve glycemic control and promote weight loss in individuals with type 2 diabetes mellitus (T2DM) and/or obesity. However, there is a paucity of evidence regarding GLP-1 agonist use in people with cystic fibrosis (pwCF). We present 11 people with CF (males: 3, females: 7; age range 24–47; BMI range 25.7–43.7) treated with GLP-1 agonists (semaglutide: 9,tirzepatide: 2) for variable duration (1–50 months). All experienced weight loss on GLP- 1 agonist therapy (median change in weight = -7.2 kg; change in BMI [kg/m2] = -0.9 to -8.1). Eight pwCF showed improvement in percent predicted forced expiratory volume in 1 second (ppFEV1) [change = -5 to + 18] and nine pwCF showed improvement in percent predicted forced vital capacity (ppFVC) [change= +1 to + 26]. Of the 7 pwCF with CFRD, all reduced their insulin quantity (mean, 31.5 % decrease in total daily insulin dose), and glucose time in range improved for most (mean, +11 % increase from baseline). Four pwCF stopped using GLP-1 agonists: 2 due to severe nausea/vomiting, 1 due to lack of perceived benefit, and 1 due to change in insurance coverage. This report is the largest published series to date of pwCF treated with GLP-1 agonist therapy. With the addition of GLP-1 agonists, all individuals experienced weight loss and a reduction in daily insulin dose, and most had improvement in pulmonary function. Future multi-center studies are needed to corroborate the efficacy and safety of these agents in the CF population.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 40-46"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142107829","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Self-reported chronic therapy use after 24-weeks of follow-up by participants who completed the simplify randomized, controlled trial","authors":"Alex H. Gifford ,&nbsp;Katherine Odem-Davis ,&nbsp;Margaret Kloster ,&nbsp;Brian P. O'Sullivan ,&nbsp;Gregory J. Omlor ,&nbsp;Susan L. Millard ,&nbsp;John P. Clancy ,&nbsp;Gregory S. Sawicki ,&nbsp;Kristin Riekert ,&nbsp;Nicole Mayer-Hamblett ,&nbsp;David P. Nichols ,&nbsp;SIMPLIFY Study Group","doi":"10.1016/j.jcf.2024.08.008","DOIUrl":"10.1016/j.jcf.2024.08.008","url":null,"abstract":"<div><h3>Background</h3><div>Highly effective CFTR modulator therapy (HEMT) has improved the health of many people with cystic fibrosis (pwCF), offering opportunities to discontinue burdensome therapies. SIMPLIFY included randomized, controlled trials that confirmed non-inferiority of discontinuing versus continuing dornase alfa (DA) or hypertonic saline (HS) for 6 weeks in pwCF on HEMT. In this study of post-trial treatment use by SIMPLIFY participants, we hypothesized that randomization to discontinue DA or HS during the trial would be associated with a higher likelihood of non-use of each medication during follow-up.</div></div><div><h3>Methods</h3><div>We electronically surveyed SIMPLIFY participants every 4 weeks for 24 weeks after trial completion but before the main trial results were publicly disclosed. We asked them how often they used medications during the previous week. We estimated covariate-adjusted odds ratios (ORs) of DA or HS non-use by logistic regression with generalized estimating equations.</div></div><div><h3>Results</h3><div>After exclusions mostly due to lack of any surveys, 472 participants were included in the analysis population, 181 from the HS trial and 291 from the DA trial. Approximately half of the analysis population completed all six surveys. At every month of follow-up in both trials, the percentage of individuals reporting non-use of DA or HS during the previous week was greater among those randomized to discontinue therapy. Among participants with responses at 24 weeks, 30/122 (24.6 %) in the HS trial and 79/222 (35.6 %) in the DA trial reported non-use of the respective study medication. After adjusting for covariates, participants randomized to discontinue DA were 8.7-times (95 % CI: 4.3–17.7) more likely to not use DA during follow-up than those randomized to continue DA, and participants randomized to discontinue HS were 5.2-times (95 % CI: 2.1–12.8) more likely to not use HS during follow-up compared to those randomized to continue.</div></div><div><h3>Conclusions</h3><div>In healthy pwCF on ETI, randomization to discontinue DA or HS during SIMPLIFY was associated with greater odds of not using each medication after the trial compared to randomization to continue. These findings suggest that participation in a treatment discontinuation trial can influence participants’ post-trial treatment decisions. This possibility may be relevant during discussions about research participation and clinical care.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 91-97"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142288303","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development of metabolic syndrome in people with Cystic Fibrosis one year after exposure to elexacaftor-tezacaftor-ivacaftor","authors":"Gregory A. Ratti ,&nbsp;Hannah Smith ,&nbsp;Sasan Mirfakhraee ,&nbsp;Joan Reisch ,&nbsp;Leah Cohen ,&nbsp;Raksha Jain ,&nbsp;James D. Finklea","doi":"10.1016/j.jcf.2024.09.022","DOIUrl":"10.1016/j.jcf.2024.09.022","url":null,"abstract":"<div><h3>Background</h3><div>The constellation of hypertension, truncal obesity, impaired fasting glucose, low high-density lipoprotein, and hypertriglyceridemia is known as metabolic syndrome (MetSyn) and is associated with cardiovascular and other diseases. Elexacaftor-tezacaftor-ivacaftor (ETI) in people with cystic fibrosis (pwCF) is associated with weight gain but effects on cardiovascular risk are unknown. This study sought to investigate ETI exposure and risk for development of MetSyn in pwCF.</div></div><div><h3>Methods</h3><div>A prospective cohort study including pwCF ≥ 18 years old exposed to ETI was performed. All data for calculating MetSyn was collected from the electronic medical record at initiation and 1 year ± 3 months after starting ETI. A total of 152 pwCF exposed to ETI and 34 pwCF never exposed to CF transmembrane conductance regulator modulators were included in the analysis. Changes to hypertension classification was also examined over this period.</div></div><div><h3>Results</h3><div>After 1 year of ETI there was an increase in MetSyn from 13 to 30 pwCF, <em>p</em> &lt; 0.0001. No new cases of MetSyn were seen in the group not exposed to ETI. After 1 year of ETI, more people met criteria for class 1 (BP 130–139/90–99 mm Hg) or class 2 hypertension (BP ≥140/≥90 mm Hg) regardless of prior modulator exposure, <em>p</em> &lt; 0.0001.</div></div><div><h3>Conclusions</h3><div>Exposure to ETI for 1 year resulted in an increased number of cases of MetSyn. There was an increased incidence of hypertension associated with ETI exposure. Additional studies are needed to further examine this trend and to determine if these changes will translate to cardiovascular complications over time.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 47-52"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142466564","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Exogenous insulin does not reduce protein catabolism in pre-diabetic cystic fibrosis patients: A randomized clinical trial","authors":"Michele Schiavon ,&nbsp;Claudio Cobelli ,&nbsp;K. Sreekumaran Nair ,&nbsp;Katherine Klaus ,&nbsp;Gianna Toffolo ,&nbsp;Lin Zhang ,&nbsp;Antoinette Moran","doi":"10.1016/j.jcf.2024.10.005","DOIUrl":"10.1016/j.jcf.2024.10.005","url":null,"abstract":"<div><h3>Background</h3><div>Cystic Fibrosis (CF) patients historically suffered from undernutrition, infection and inflammation. Insulin insufficiency-related protein catabolism further compromised health. We aimed to determine whether insulin improves protein catabolism in CF youth with abnormal glucose tolerance (AGT).</div></div><div><h3>Methods</h3><div>This double-masked, placebo-controlled trial in CF youth age 10–25 with AGT who were in their usual state of health used triple-tracer stable-isotope methodology to measure protein turnover during a baseline test meal and after four weeks of insulin/placebo treatment. Healthy controls were assessed once. CF patients were randomized 1:1:1 to once-daily long-acting insulin (0.25 U/kg/d), three-times daily rapid-acting insulin (0.5 U/15gr carbohydrate), or injectable placebo.</div></div><div><h3>Results</h3><div>Thirty CF patients completed the study. There were no differences in any measure of protein turnover between insulin- and placebo-treated subjects, including endogenous protein breakdown (primary study endpoint). In contrast to earlier studies, protein turnover in the 37 CF patients who completed the baseline meal was normal compared to 20 healthy controls. Meal isotope appeared in plasma earlier in CF than controls, suggesting more rapid gut emptying. The study was interrupted by the pandemic; futility analysis led to study discontinuation before the planned remaining 15 CF patients were studied.</div></div><div><h3>Conclusions</h3><div>Recent advances in CF have led to remarkable clinical improvements. In this study, CF youth with AGT had normal protein catabolism at baseline. Pre-meal or daily basal insulin therapy, while safe and well tolerated, did not significantly enhance protein turnover and does not appear to be necessary in clinically stable patients prior to development of CFRD.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 57-65"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11788023/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142620390","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Learning from the CFTR modulator baby boom","authors":"Raksha Jain ,&nbsp;Jennifer L. Goralski","doi":"10.1016/j.jcf.2024.12.003","DOIUrl":"10.1016/j.jcf.2024.12.003","url":null,"abstract":"","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 3-4"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142965140","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}