AnatomyOrganismsDiseasesChemicals and DrugsAnalytical, Diagnostic and Therapeutic Techniques and EquipmentPhenomena and ProcessesDisciplines and OccupationsAnthropology, Education, Sociology and Social PhenomenaInformation ScienceNamed GroupsHealth CareGeographicals
Cystic FibrosisPseudomonas InfectionsPseudomonas aeruginosaCystic Fibrosis Transmembrane Conductance RegulatorSputumBurkholderia InfectionsAchromobacter denitrificansBurkholderia cepaciaFibrosisBurkholderia cepacia complexTobramycinExocrine Pancreatic InsufficiencyBurkholderia cenocepaciaPancreatic ExtractsAlginatesAnti-Bacterial AgentsStenotrophomonas maltophiliaPancreatinSweatAspergillosis, Allergic BronchopulmonaryGram-Negative Bacterial InfectionsDrainage, PosturalForced Expiratory VolumeHexuronic AcidsGlucuronic AcidRespiratory SystemLungLung DiseasesBronchopneumoniaRespiratory Function TestsColistinAchromobacterExpectorantsMutationBurkholderiaLung TransplantationStreptococcus constellatusBronchiectasisCampingAdministration, InhalationGenotypeNasal MucosaIdiopathic Pulmonary FibrosisMicrobial Sensitivity TestsHome Infusion TherapyChloridesLeukocyte ElastasePercussionCeftazidimeRespiratory Tract InfectionsDNA, BacterialElectrophoresis, Gel, Pulsed-FieldBacterial Typing TechniquesPolymerase Chain ReactionPhenotypeMucusBiofilmsDNA FingerprintingMeconiumPneumonia, BacterialVital CapacityAzithromycinBacterial ProteinsRandom Amplified Polymorphic DNA TechniqueRalstoniaRespiratory MucosaBronchiMice, Inbred CFTRDrug Resistance, BacterialHomozygoteStaphylococcus aureusMolecular Sequence DataPyocyanineDrug IncompatibilityChronic DiseaseMalabsorption SyndromesRespiratory TherapyAspergillus fumigatusNebulizers and VaporizersBreath TestsEndomyocardial FibrosisOpportunistic InfectionsPancreatic ElastaseEpithelial CellsPolymyxinsGene Expression Regulation, BacterialAerosolsNutritional StatusPseudomonasDrug Resistance, MicrobialStaphylococcal InfectionsBronchoalveolar Lavage FluidAgarTime FactorsSensitivity and SpecificityLiver CirrhosisCiprofloxacinPolymorphism, Restriction Fragment LengthPancreasChloride Channels
LungsCFTRNorth American Cystic Fibrosis Conference2019PseudomonasEuropean Cystic Fibrosis SocietyGeneticLiving With Cystic FibrosisRespiratoryInfections in cystic fibrosis patientsPulmonaryChild with cystic fibrosisInfants with cystic fibrosisIndividuals with cystic fibrosisAffects2023IvacaftorAutosomal recessiveMutationGeneInflammationInfectionPancreasSputum samplesStage biopharmaceutical companyTreatmentsTrikaftaBacteriaBronchiectasisBacterialSecretionsMicrobiotaPeopleChronicAeruginosaCarriersDiagnosisLung TransplantationBurkholderiaRisk FactorsMucus and sweat30,000Clinical trialAdultPathogenHepaticAntibioticPediatricProgressionExacerbationsAdherenceFoundationStaphylococcusTherapiesTreatmentIsolatesMolecularPancreaticSweat
Lungs36
  • Cystic fibrosis results from a genetic mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which produces a faulty ion channel that prevents chloride ions from exiting cells in the lungs and other organs. (nationaljewish.org)
  • Patients suffer repeated infections of the lungs, which result in lung damage over time and eventually death. (nationaljewish.org)
  • Their study, published in Genome Research this week, provides the first comprehensive genome-phenome analyses of B. cenocepacia infection in cystic fibrosis lungs. (drexel.edu)
  • The cocktail contains four bacteriophages (viruses that kill or neutralize bacteria) that target Pseudomonas aeruginosa , a multidrug-resistant bacterium that colonizes the lungs of CF patients and is the most common cause of CF exacerbations. (umn.edu)
  • This designation is a major boost to our efforts to make this drug available as quickly as possible to cystic fibrosis patients who currently suffer from fungal infections in their lungs, and from the allergic reactions they experience because of the fungal infections," says Pulmatrix CEO Robert Clarke , PhD. (prnewswire.com)
  • Pulmatrix's PUR1900 combines an existing antifungal drug, itraconazole, with the company's innovative dry powder iSPERSE technology , enabling patients to easily inhale the drug deep into their lungs where it's needed. (prnewswire.com)
  • Our technology delivers the drug directly to the lungs," explains Pulmatrix's Chief Scientific Officer, David L. Hava , PhD. "That significantly reduces the risks of side effects and drug-drug interactions, bringing great benefits to patients . (prnewswire.com)
  • Pulmatrix's product candidates are based on iSPERSE™, its proprietary dry powder delivery platform, which seeks to improve therapeutic delivery to the lungs by maximizing local concentrations and reducing systemic side effects to improve patient outcomes. (prnewswire.com)
  • We have already demonstrated a significant improvement in the blood glucose levels in patients with metabolic syndrome with Type 2 diabetes treated with PBI-4050, and a significant reduction of fibrosis in the lungs, pancreas and liver in multiple animal models. (newswire.ca)
  • We believe that the unique mode of action of PBI-4050 could indeed provide much relief for the CF patients by reducing the level of fibrosis in their lungs and pancreas and by improving the production of insulin" concluded Dr. Moran. (newswire.ca)
  • Cystic fibrosis (CF), also known as mucoviscidosis, is a life-threatening, genetic disease that affects especially the lungs but also the pancreas, liver, kidneys, and gut. (newswire.ca)
  • Cystic fibrosis (CF) is a rare, progressive disorder affecting the lungs, pancreas, and other organs, and has a significant burden on patients' functioning and quality of life (QoL). (ispor.org)
  • Cystic fibrosis (CF) is a rare genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys, and intestine. (wikipedia.org)
  • NEW YORK (GenomeWeb) - Isolation of bacteria within different regions of the lungs of cystic fibrosis patients drives their diversification, according to a phylogenetic study appearing in Cell Host & Microbe today . (genomeweb.com)
  • Researchers from the University of Washington School of Medicine dissected 10 pairs of lungs from cystic fibrosis patients - obtained after lung transplantation - and examined some 12,000 isolates of Pseudomona aeruginosa inhabiting various lung regions. (genomeweb.com)
  • Jorth and his colleagues sampled both left and right lungs from 10 cystic fibrosis patients at three sites: the upper, middle, and lower lobe airways. (genomeweb.com)
  • The researchers used phenotypic data to classify the isolates into subpopulations, and found that each patient harbored between 12 and 40 bacterial subpopulations in their lungs and that each lobar region contained between one and three of those sub-populations. (genomeweb.com)
  • Based on their finding of strong signals of genetic compartmentalization, regional isolates with similar protein expression profiles, and spatially distinct distribution of bacterial phenotypes, the researchers said that their study indicates that isolation in different lung regions helped drive the diversification of P. aeruginosa in the lungs of cystic fibrosis patients. (genomeweb.com)
  • A defective gene causes thick, sticky mucus to build up in the lungs of patients with cystic fibrosis (CF). There, it traps bacteria, causing patients to develop frequent lung infections that progressively damage these vital organs and impair patients' ability to breathe. (mecfa.org)
  • Thirty days after patients completed antibiotic therapy, when their lungs' bacterial flora had some time to bounce back. (mecfa.org)
  • Lung ventilation heterogeneity due to the obstruction of small airways is an early and potentially reversible step in the progression of cystic fibrosis (CF). Individually, multiple breath washout (MBW), hyperpolarised (HP) gas ventilation-MRI and conventional structural lung imaging by CT have all been shown to be sensitive to early changes in the lungs before spirometry. (bmj.com)
  • Cystic Fibrosis, commonly known as CF, is a life-threatening, lethal genetic disease that mostly affects the lungs and digestive system (O'Sullivan & Freedman, 2009). (bartleby.com)
  • 12] Unusually viscous emissions in the airway of the lungs and in ducts of the pancreas in people with cystic fibrosis cause hindrances that prompt aggravation, tissue harm and destruction of both organ systems. (bartleby.com)
  • Cystic Fibrosis is a life threatening condition that causes severe damage to the lungs and digestive system. (bartleby.com)
  • The pileup affects the fluid balance of tissue, resulting in the thick mucus that characterizes the lungs of CF patients. (biotechprimer.com)
  • Cystic fibrosis (CF) is an inherited life-threatening disorder that damages the lungs and digestive system. (abilitymagazine.com)
  • It describes a potential strategy for treating cystic fibrosis (CF) - an inherited, fatal disease that affects 30,000 Americans - by using slightly acidified sodium nitrite, a common food preservative, to kill antibiotic-resistant bacteria that thrive in the mucous-clogged lungs of CF patients. (bnl.gov)
  • CINCINNATI-Researchers led by a University of Cincinnati (UC) scientist say they have discovered what might be the "Achilles' heel" of a dangerous organism that lives in the lungs of cystic fibrosis patients-a fatal flaw that leaves the organism vulnerable to destruction by a common food preservative. (bnl.gov)
  • The new study suggests, however, that a mutation-known as mucA-in the organism also represents a fatal flaw that could help physicians clear the characteristic "goop" from the lungs of advanced cystic fibrosis patients. (bnl.gov)
  • During the chronic form of cystic fibrosis," Dr. Hassett said, "the mutated form of the organism, combined with the immune system's attempts to fight it off, wreaks havoc in the lungs. (bnl.gov)
  • Using anonymised data hosted by the Secure Anonymised Information Linkage (SAIL) Databank , Robert is investigating the degree to which carriers of cystic fibrosis, a genetic condition which causes severe damage to the lungs, digestive system and other organs, are at risk of developing conditions related to the disease, such as diabetes, bronchiectasis and pancreatitis. (saildatabank.com)
  • Cystic fibrosis is a disease affecting the lungs and digestive system. (xshotpix.com)
  • Over time, mucus buildup and infections can lead to permanent lung damage, including the formation of scar tissue (fibrosis) and cysts in the lungs. (xshotpix.com)
  • The organs most often affected by cystic fibrosis are the lungs and the pancreas, which can lead to breathing and digestive problems. (chp.edu)
  • In a person with cystic fibrosis, mucus is still able to trap bacteria, but it has trouble moving out of the lungs. (chp.edu)
  • Both of these medications work by improving the function of a protein in the body to decrease the build-up of thick mucus in the lungs and improving other symptoms of cystic fibrosis. (medlineplus.gov)
CFTR18
  • The U.S. Food and Drug Administration (FDA) has already approved a similar combination of Ivacaftor and Lumacaftor for patients who have two copies of the most common CFTR mutation, F508del. (nationaljewish.org)
  • The three-drug combination's expanded label now covers children ages 2 to 5 years who have cystic fibrosis and either a F508del mutation in the CFTR gene or a CFTR gene mutation that has been shown to be responsive to the triplet based on in vitro data. (medpagetoday.com)
  • Early intervention with CFTR modulator therapies like Trikafta can offer the potential to improve the trajectory of [cystic fibrosis] lung disease," said trial investigator Jennifer Goralski, MD, of University of North Carolina School of Medicine in Chapel Hill, in a statement. (medpagetoday.com)
  • Based on the results of two randomized trials, the triplet combination first won approval in 2019 for adolescents and adults with susceptible CFTR gene mutations, a group representing an estimated 90% of the population of patients with cystic fibrosis at that time. (medpagetoday.com)
  • Dysfunction of cystic fibrosis transmembrane regulator (CFTR) in the intestine also promotes changes in local microbiota such as small intestinal bacterial overgrowth (SIBO), which is common in CF. We evaluated whether therapy with the CFTR modulator combination lumacaftor/ivacaftor (luma/iva) has a beneficial impact on SIBO as measured by breath testing (BT). (nih.gov)
  • METHODS Polymorphisms in the TGF-β 1 gene defining amino acids of codons 10 and 25 were determined by ARMS-PCR using DNA stored on 171 Caucasian patients who were homozygous for the ΔF508 mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. (bmj.com)
  • Despite advances in the medical management of cystic fibrosis over the last three decades, there are still a number of unexplained differences in its clinical course, especially in the rate of development of respiratory failure of patients who appear to have the same CFTR genotype, degree of bacterial colonisation, and compliance with medication. (bmj.com)
  • Severe liver disease distinguished by cirrhosis with portal hypertension may be developed by a small fraction about 3 - 5 percent with cystic fibrosis (CF). Prior study has claimed that genetic variability that is not related with the cystic fibrosis transmembrane conductance regulator (CFTR) gene may add to the danger for severe liver disease. (healthjockey.com)
  • It is caused by the presence of mutations in both copies (alleles) of the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein. (wikipedia.org)
  • It is caused by a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) resulting in abnormal membrane osmolarity. (brighton.ac.uk)
  • An individual with cystic fibrosis has a specific mutation in a gene and protein called cystic fibrosis transmembrane conductance regulator (CFTR). (bartleby.com)
  • CF hinges on one of several possible mutations in the gene that encodes the "cystic fibrosis transmembrane conductance regulator" (CFTR) protein. (biotechprimer.com)
  • Kalydeco alone doesn't help patients whose mutation prevents CFTR from reaching the cell surface. (biotechprimer.com)
  • Cystic fibrosis (CF) is an autosomal recessive condition caused by the mutation of the cystic fibrosis trans-membrane regulator gene (CFTR) on chromosome 7. (who.int)
  • Cystic fibrosis, which affects about 30,000 people in the United States, mostly Caucasians of north European origin, is an inherited disease caused by a defect in a gene called the cystic fibrosis transmembrane conductance regulator (CFTR). (bnl.gov)
  • 5 Called the cystic fibrosis transmembrane conductance regulator (CFTR) gene, this mutation prevents the CFTR protein from working properly. (xshotpix.com)
  • Lumacaftor is in a class of medications called cystic fibrosis transmembrane conductance regulator (CFTR) correctors. (medlineplus.gov)
  • Ivacaftor is in a class of medications called cystic fibrosis transmembrane conductance regulator (CFTR) potentiators. (medlineplus.gov)
North American Cystic Fibrosis Conference3
  • We have been directly working on this for many years at our center, ever since one of the drugs that now is also one-third of the medication that comprises Trikafta was first studied in 2011," says Green, who will attend the North American Cystic Fibrosis Conference this week in Nashville. (hopkinsmedicine.org)
  • As Michelle Prickett showed during plenary 1 of this year's North American Cystic Fibrosis Conference, CF care teams adapted to provide care and keep us safe. (cff.org)
  • After watching the second plenary of the 2021 North American Cystic Fibrosis Conference, I was incredibly impressed by the level of support the Cystic Fibrosis Foundation is providing to the development of the next generation of therapeutics. (cff.org)
20191
  • Outbreak of Pandoraea commovens Infections among Non-Cystic Fibrosis Intensive Care Patients, Germany, 2019-2021. (cdc.gov)
Pseudomonas13
European Cystic Fibrosis Society1
Genetic14
  • Cystic fibrosis (CF) is the most common fatal genetic disorder in the world. (drexel.edu)
  • A genetic disorder which is known to be a hereditary disease of the secretory glands, as well as the glands that makes mucus and sweat is called as cystic fibrosis. (healthjockey.com)
  • A genetic analysis now points out that a particular gene variation in patients suffering from cystic fibrosis could considerably add to their possibility of developing severe liver disease. (healthjockey.com)
  • The preliminary study apparently evaluated genetic variations in the candidate genes in about 124 people with CFLD and in 843 control patients without CFLD. (healthjockey.com)
  • The 14-year-old from Sarasota has cystic fibrosis , a genetic disease that can cause persistent lung infections and limits the ability to breathe over time. (hopkinsmedicine.org)
  • Using that sequencing data, the researchers also examined the genetic diversity of the P. aeruginosa from these three patients. (genomeweb.com)
  • Non-resolving inflammation is the main mechanism of morbidity and mortality among patients suffering from cystic fibrosis (CF), the most common life-threatening human genetic disease. (frontiersin.org)
  • Most patients with this progressive genetic disorder die by the fourth decade of life. (mecfa.org)
  • Cystic Fibrosis is a genetic disease which is progressive and limits breathing ability. (bartleby.com)
  • The suspected diagnosis of cystic fibrosis was confirmed with a sweat chloride level of 120 mmol/L and homo-zygozity for the Delta F 508 gene on genetic studies. (who.int)
  • We want to understand if particular cystic fibrosis genetic mutations contribute to increased risks of carriers developing associated conditions. (saildatabank.com)
  • Influence of genetic variation of the ß2-adrenergic receptor on lung diffusion in patients with cystic fibrosis. (bvsalud.org)
  • Cystic fibrosis (CF) is an inherited (genetic) condition found in children that affects the way salt and water move in and out of cells. (chp.edu)
  • Because there are hundreds of specific cystic fibrosis gene mutations (not all of which are known), genetic testing for cystic fibrosis is not 100 percent sensitive. (chp.edu)
Living With Cystic Fibrosis3
  • As of 2022, approximately 40,000 people in the U.S. are living with cystic fibrosis. (medpagetoday.com)
  • A drug tested at Children's Hospital Colorado and other sites around the country proved effective and is expected to help 90 percent of people living with cystic fibrosis. (cuanschutz.edu)
  • If approached thoughtfully, the new movie Five Feet Apart may provide an opportunity for constructive conversations about living with cystic fibrosis, and help raise awareness around the disease. (chop.edu)
Respiratory14
  • Patients taking the ivacaftor-tezacaftor combination also experienced fewer respiratory adverse events, including respiratory infections, cough, headache and fatigue, than did those taking placebo. (nationaljewish.org)
  • Other gram-negative glucose nonfermenters, such as Achromobacter xylosoxidans , B. gla-dioli , Ralstonia pickettii , and Stenotrophomonas maltophilia are also occasionally reco-vered from respiratory samples from CF patients, but their pathogenic importance remains to be clarified ( 2 - 4 ). (cdc.gov)
  • In 1 of these studies, the gram-negative nonfermentative species Inquilinus limosus was newly described in respiratory secretions of 8 patients with CF in the United States ( 8 ). (cdc.gov)
  • To detect unusual gram-negative microbes in respiratory samples, we screened all patients attending the CF clinic of the University Hospital, Ulm, Germany, from May 2002 to September 2004 (N = 85 patients). (cdc.gov)
  • Strain A was isolated from a 17-year-old male patient with CF with persistent colonization of the respiratory tract since childhood with S. aureus , P. aeruginosa , including the mucoid variant, and H. influenzae . (cdc.gov)
  • Cystic fibrosis (CF) is a disease of exocrine gland function that involves multiple organ systems but chiefly results in chronic respiratory infections, pancreatic enzyme insufficiency, and associated complications in untreated patients. (medscape.com)
  • Cystic fibrosis affects the body by producing thick mucus in varying organ systems which can cause chronic sinus tract infections, respiratory infections, malabsorption disorders, increased metabolic demand, pancreatic dysfunction and potential sterility in males. (hopkinsmedicine.org)
  • When people with cystic fibrosis get a respiratory virus, they may get sicker because of their lung disease. (chop.edu)
  • The primary cause of morbidity and death in people with cystic fibrosis is progressive lung disease, which eventually leads to respiratory failure. (wikipedia.org)
  • Mucus hypersecretion with abnormal mucus consistency and reduced mucociliary clearance occurs in respiratory diseases like cystic fibrosis (CF), respiratory distress syndrome (RDS) of the newborn, chronic obstructive pulmonary disease, asthma and also in heavy smokers. (karger.com)
  • Affecting the airways and many other vital organs and processes, cystic fibrosis is chronic, progressive and ultimately fatal, mostly as a result of respiratory failure. (bnl.gov)
  • The prospective study, "Early respiratory viral infections in infants with cystic fibrosis," was published in the Journal of Cystic Fibrosis. (xshotpix.com)
  • Discrepancy in MALDI-TOF MS identification of uncommon Gram-negative bacteria from lower respiratory secretions in patients with cystic fibrosis. (cdc.gov)
  • Time between collection and storage significantly influences bacterial sequence composition in sputum samples from cystic fibrosis respiratory infections. (cdc.gov)
Infections in cystic fibrosis patients2
  • Scientists have made new headway in understanding how a deadly pathogen evolves during chronic lung infections in cystic fibrosis patients. (drexel.edu)
  • PULM ) announced today that the U.S. Food & Drug Administration has granted the company's request for orphan drug status for its inhaled drug for treating pulmonary fungal infections in cystic fibrosis patients, named PUR1900. (prnewswire.com)
Pulmonary15
  • Alteration of the airway microbiota is a hallmark of cystic fibrosis (CF) pulmonary disease. (nih.gov)
  • The estimated addressable market for improved antifungal treatments for CF is in the tens of thousands of patients per year," says Dr. Clarke, "but the inhaled drug could also find much larger markets treating pulmonary fungal infections and other immunocompromised patients that could expand the addressable market to millions of patients per year. (prnewswire.com)
  • The Company's proprietary product pipeline is focused on advancing treatments for rare diseases, including PUR1900, an inhaled anti-fungal for patients with cystic fibrosis (CF). In addition, Pulmatrix is pursuing opportunities in major pulmonary diseases through collaborations, including PUR0200, a branded generic in clinical development for chronic obstructive pulmonary disease (COPD). (prnewswire.com)
  • Patients with cystic fibrosis (CF) show decreased plasma concentrations of antioxidants due to malabsorption of lipid soluble vitamins and consumption by chronic pulmonary inflammation. (hindawi.com)
  • Chronic pulmonary infection of CF patients results in a chronic activation of immune cells, especially neutrophilic granulocytes, leading to a permanent production of free radicals and lipid peroxidation (LPO) products, thus triggering inflammation [ 3 - 6 ]. (hindawi.com)
  • In a number of studies, β -carotene supplementation in CF patients led to positive effects such as increase of total antioxidant capacity and decrease of pulmonary exacerbations [ 15 - 17 ]. (hindawi.com)
  • This study was undertaken to examine the association between TGF-β 1 gene polymorphisms and the development of pulmonary dysfunction in patients with cystic fibrosis. (bmj.com)
  • DISCUSSION TGF-β 1 genotypes may have a role in mediating pulmonary dysfunction in patients with cystic fibrosis. (bmj.com)
  • 12 In this study we have investigated the correlation between high TGF-β 1 production and pulmonary dysfunction in cystic fibrosis by determination of polymorphisms in the TGF-β 1 gene. (bmj.com)
  • Pulmonary involvement (see the image below) occurs in 90% of patients surviving the neonatal period. (medscape.com)
  • Clement Ren, MD, MBA, joined the Division of Pulmonary and Sleep Medicine in July and will lead the Cystic Fibrosis Center. (chop.edu)
  • But how antibiotic resistance impacts the relationship between lung bacterial diversity and CF patients' pulmonary function has been unknown. (mecfa.org)
  • Their findings suggest that the presence of multidrug resistant bacteria in the airways of patients with CF is associated with decreased microbial diversity and decreased pulmonary function. (mecfa.org)
  • Although heavy antibiotic use probably contributed to both the antibiotic resistance and lowered microbial diversity, Dr. Hahn says, the answer isn't to reduce use of these drugs: They're necessary to help patients with CF recover after each bout with pulmonary exacerbations. (mecfa.org)
  • P. aeruginosa is the dominant pathogen in end-stage CF lung disease, and chronic infection with P. aeruginosa is correlated with more severe reductions in pulmonary function measures (27) and mortality in CF patients. (xshotpix.com)
Child with cystic fibrosis2
Infants with cystic fibrosis1
  • Newborns and infants with cystic fibrosis tend to have frequent, large, greasy stools (a result of malabsorption) and are underweight for their age. (wikipedia.org)
Individuals with cystic fibrosis2
  • Years ago, individuals with cystic fibrosis would rarely live beyond his or her childhood, but now many individuals with the disease are living well into their adulthood (Falvo, 2014). (bartleby.com)
  • Longitudinal sampling of the lung microbiota in individuals with cystic fibrosis. (cdc.gov)
Affects3
  • The trial aims to determine whether the treatment is safe and if it reduces the amount of bacteria in trial volunteers, with investigators gathering data on safety and microbiologic activity, how the phages function in the body, how the therapy affects patients' lung function, and it s impact on overall quality of life. (umn.edu)
  • Cystic fibrosis (CF) affects between 1in 2000 and 1 in 4500 births in caucasians of European descent. (brighton.ac.uk)
  • RATIONALE Cystic fibrosis (CF) is a disease that adversely affects the lung resulting in a reduction in lung diffusion . (bvsalud.org)
20231
  • To mark Jeans for Genes Week 2023 (18-24 September), Robert Maddison, a researcher funded by Health and Care Research Wales Faculty, discusses his study into the health of carriers of cystic fibrosis. (saildatabank.com)
Ivacaftor5
  • Ivacaftor, already approved for treatment of certain forms of cystic fibrosis, helps keep the faulty ion channel on the cell surface open so chloride and other ions can exit the cell. (nationaljewish.org)
  • In the current study, Dr. Taylor-Cousar and colleagues from 91 study sites in the United States, Canada and Europe, evaluated 475 patients with two copies of 508del who took the ivacaftor-tezacaftor combination or a placebo for 24 weeks. (nationaljewish.org)
  • The FDA expanded the indication for elexacaftor, tezacaftor, and ivacaftor (Trikafta) in cystic fibrosis to include even younger children, maker Vertex Pharmaceuticals announced . (medpagetoday.com)
  • Lumacaftor and ivacaftor is used to treat certain types of cystic fibrosis (an inborn disease that causes problems with breathing, digestion, and reproduction) in adults and children 1 year of age and older. (medlineplus.gov)
  • Lumacaftor and ivacaftor controls cystic fibrosis but does not cure it. (medlineplus.gov)
Autosomal recessive3
  • Cystic fibrosis is inherited in an autosomal recessive manner. (wikipedia.org)
  • Disability Overview Cystic fibrosis is the most common autosomal recessive disorder in Caucasian's with an incidence rate of 1 in every 2,500 births. (bartleby.com)
  • Cystic fibrosis is an existence restricting autosomal recessive disorder that influences 70,000 people around the world. (bartleby.com)
Mutation2
  • It's a big deal that there's finally something that can reach patients with this mutation," says Sam's mother, Lisa. (hopkinsmedicine.org)
  • We have a center of 165 patients, and of those, more than 80 are over the age of 12 and about 70 would qualify based on the type of CF mutation they have. (hopkinsmedicine.org)
Gene10
  • We expected, based on anecdotal observations and single strain reports, that the genome of B. cenocepacia was flexible, but we had no idea of the scope and scale of how promiscuous the gene content and genome architecture would be in a modest-sized patient cohort," said co-corresponding author, Corey Nislow, PhD, an assistant professor at the University of British Columbia. (drexel.edu)
  • Nearly 1 in every 30 Americans are a carrier of the recessive gene that leads to CF (About Cystic Fibrosis, n.d. (bartleby.com)
  • With more than 2,000 identified mutations of the CF gene, cystic fibrosis is a complex condition with significant challenges to find an effective way to treat this incurable disease. (politicshome.com)
  • How is the cystic fibrosis ( CF ) gene inherited? (xshotpix.com)
  • The gene that causes cystic fibrosis is recessive. (chp.edu)
  • This means that in order to have cystic fibrosis, children must inherit two copies of the gene, one from each parent. (chp.edu)
  • Parents who carry the cystic fibrosis gene are often healthy and have no symptoms of disease, and yet are still likely to pass it on to their children. (chp.edu)
  • In fact, it's estimated that as many as 10 million people may be carriers of a cystic fibrosis gene and not know it. (chp.edu)
  • The cystic fibrosis gene is most common in Caucasians of northern European descent. (chp.edu)
  • however, it's not possible to identify every person who carries a gene for cystic fibrosis. (chp.edu)
Inflammation2
Infection5
  • One patient each developed pre-eclampsia after Omicron infection, delivered via caesarean at 36 weeks' gestation due to UGR after wild-type infection, and miscarried at 19 weeks due to UGR after Alpha infection. (umn.edu)
  • According to the Cystic Fibrosis Foundation , people with CF need to keep a distance of 6 feet between each other and between those with an infection, including a cold or flu. (medicalnewstoday.com)
  • CHOC uses highest standard of infection control by following the Cystic Fibrosis Foundation infection control recommended guidelines. (choc.org)
  • Is cystic fibrosis a viral infection? (xshotpix.com)
  • Which bacteria causes the greatest infection burden in cystic fibrosis patients? (xshotpix.com)
Pancreas4
  • The name "cystic fibrosis" refers to the characteristic fibrosis and cysts that form within the pancreas. (wikipedia.org)
  • The company pointed out that according to the Cystic Fibrosis Foundation there are over 30,000 patients in the U.S. with EPI caused by CF and the National Pancreas Foundation lists that there are around 90,000 people in the U.S. with EPI caused by chronic pancreatitis. (streetwisereports.com)
  • In a person with cystic fibrosis, the thick, sticky mucus blocks ducts (or paths) between the pancreas and the intestines . (chp.edu)
  • Because the pancreas controls the level of sugar in the blood, a small percentage of people with cystic fibrosis may also develop type-one diabetes (formerly called juvenile or insulin-dependent diabetes). (chp.edu)
Sputum samples1
Stage biopharmaceutical company1
  • Clinical stage biopharmaceutical company AzurRx BioPharma Inc. (AZRX:NASDAQ) , which focuses its efforts on developing targeted, non-systemic recombinant therapies for treating gastrointestinal (GI) diseases, yesterday announced that "the first two patients have been dosed in the Phase 2b OPTION 2 extension study of MS1819 using immediate release capsules for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF). (streetwisereports.com)
Treatments9
  • A need for alternative treatments remains for EPI patients who are unable to maintain appropriate nutritional health, especially those who seek soluble or non-porcine therapeutic options" said Michael Konstan, MD, Vice Dean for Translational Research at Case Western Reserve University School of Medicine. (einpresswire.com)
  • Over the 18-month study period, these patients made multiple visits for exacerbations and antibiotic treatments, leading to samples from 19 patient encounters overall. (mecfa.org)
  • Today, the Cystic Fibrosis Foundation announced a collaboration with Deep Science Ventures to accelerate treatments that address the underlying cause of CF for every person with the disease. (cff.org)
  • Researchers are exploring treatments that will keep people with cystic fibrosis as healthy as possible until a cure is found. (cff.org)
  • As the only Pediatric Cystic fibrosis Center in Orange County fully accredited by Cystic Fibrosis Foundation, you can rest assured that your child will receive the professional expertise, state-of-the-art diagnostic tools and cutting edge treatments to keep your child healthy. (choc.org)
  • Vertex Pharmaceuticals' (Boston, MA) innovative new drug, Trikafta, promises better health to a much broader range of people with cystic fibrosis (CF) than previous treatments. (biotechprimer.com)
  • I was diagnosed at age five, which meant I have five years of scarring, and I also have two nonsense mutations, which is a very complicated way of me saying that there weren't many treatments available for patients like myself. (abilitymagazine.com)
  • In my youth, once I was diagnosed at five, there were only one or two treatments available for CF patients at that time. (abilitymagazine.com)
  • Today, thanks to improved treatments, people with cystic fibrosis live an average of 35 years. (bnl.gov)
Trikafta4
  • Sam, a 14-year-old with cystic fibrosis, became the first patient at Johns Hopkins All Children's Hospital to take Trikafta, a new drug therapy that will help an estimated 90% of cystic fibrosis patients. (hopkinsmedicine.org)
  • The U.S. Food and Drug Administration (FDA) announced last week approval of Trikafta for patients 12 and older. (hopkinsmedicine.org)
  • Since 2012, Vertex has won the approval of four different drugs-Kalydeco, Orkambi, Symdeko, and now Trikafta-that effectively treat the underlying cause of cystic fibrosis. (biotechprimer.com)
  • However, only Trikafta promises to treat a significant majority-90%-of patients. (biotechprimer.com)
Bacteria4
  • RESULTS--B cepacia was recovered from room air during occupation by five of six patients, the number of bacteria ranging from 1 to 158 cfu/m3 (mean 32 cfu/m3). (whiterose.ac.uk)
  • The number of bacteria isolated was greater when patients were coughing. (whiterose.ac.uk)
  • People with cystic fibrosis (CF) have an increased risk of contracting and passing on harmful bacteria to other people with CF. This means people with CF need to avoid close physical contact with other people with the condition. (medicalnewstoday.com)
  • Patients who carried this or other antibiotic-resistant bacteria had significantly lower microbial diversity in their samples and more aggressive disease. (mecfa.org)
Bronchiectasis3
  • This is an observational multicentre study including 819 patients diagnosed with non-cystic fibrosis bronchiectasis using high-resolution computed tomography. (ersjournals.com)
  • With this study, we aimed to create and validate an easy-to-use multidimensional grading system that could classify the severity of non-cystic fibrosis (CF) bronchiectasis more accurately than any single variable, according to its capacity to predict the 5-year risk of all-cause mortality. (ersjournals.com)
  • Considerations in younger patients might also include bronchiectasis, congenital abnormalities, foreign-body aspiration, and cystic fibrosis. (cdc.gov)
Bacterial2
  • The researchers collected 215 bacterial samples isolated from 16 cystic fibrosis patients over a time, spanning a period of up to 20 years for each patient. (drexel.edu)
  • Their results revealed a host of bacterial species present in these patients' airways, including methicillin-resistant Staphylococcus aureus, a notoriously hard-to-treat microbe. (mecfa.org)
Secretions1
  • In people with cystic fibrosis, the secretions are abnormally thick and sticky, so that they don't move as easily. (chp.edu)
Microbiota1
  • Acquisition and adaptation of the airway microbiota in the early life of cystic fibrosis patients. (cdc.gov)
People11
  • In the amicus brief, patient and consumer groups outline the potential devastating impact for people with pre-existing conditions if the ACA were to be struck down and critical patient protections, as well as Medicaid expansion, were eliminated. (cff.org)
  • Is it safe for people with cystic fibrosis to be together? (medicalnewstoday.com)
  • The Cystic Fibrosis Foundation notes that if people with CF are having difficulty speaking with their partner about their condition, they may find it beneficial to speak with a member of their healthcare team or a therapist. (medicalnewstoday.com)
  • Lung problems are responsible for death in 80% of people with cystic fibrosis. (wikipedia.org)
  • TDN helps speed the delivery of new and better therapies to people with cystic fibrosis. (choc.org)
  • Half the people who die of cystic fibrosis are under the age of 31. (politicshome.com)
  • Robert, who received a Health and Care Research Wales Health PhD Studentship, hopes this project will drive future cystic fibrosis research and treatment to improve the lives of people who have the condition and their families in Wales and beyond. (saildatabank.com)
  • According to the Cystic Fibrosis Trust , over 500 people have been diagnosed with the condition in Wales. (saildatabank.com)
  • How many people are affected by cystic fibrosis? (xshotpix.com)
  • As a result, people with cystic fibrosis have trouble digesting food and getting the vitamins and nutrients they need from it. (chp.edu)
  • In some people with cystic fibrosis, the intestines, liver , sweat glands, and reproductive organs are also harmed. (chp.edu)
Chronic3
  • The critical patient protections in the Affordable Care Act (ACA) provide an essential lifeline for millions of Americans who suffer from serious, acute, and chronic health conditions. (cff.org)
  • The 9-year study included sites in the U.S. and 8 European countries and, with 6,088 CF patients and 4,102 parents screened, was the largest study of its kind conducted for a chronic illness. (cysticfibrosis.ca)
  • These include two Phase 2 clinical trials for its leading therapeutic candidate MS1819 for treatment of EPI in patients with CF and chronic pancreatitis. (streetwisereports.com)
Aeruginosa1
  • For instance, about 40 percent of the P. aeruginosa isolates they obtained from the right upper lobe of one patient were resistant to the antibiotic ciprofloxacin, while all right lower lobe isolates were sensitive to it. (genomeweb.com)
Carriers4
  • Robert said: "It has long been thought that carriers of cystic fibrosis are just as healthy as the general population. (saildatabank.com)
  • However, recent research has indicated that carriers of the disease may be at elevated risk of cystic fibrosis associated conditions. (saildatabank.com)
  • Ultimately, we want the best information for cystic fibrosis carriers and their healthcare professionals to potentially detect and treat these conditions early. (saildatabank.com)
  • Robert is using anonymised Welsh hospital, GP and census records to understand what health conditions cystic fibrosis carriers are developing, then compare those results with the cystic fibrosis patient community and the general population. (saildatabank.com)
Diagnosis1
  • Sensitivity was defined as the presence of an abnormal measurement (ie, not within the normal range for that measurement) in the presence of the diagnosis of CF. This assumes that all patients should have some abnormality in at least one measurement, which may not be true in this population with very mild clinical expression of CF, and therefore provides a conservative estimation of sensitivity of the lung assessments. (bmj.com)
Lung Transplantation3
  • 9 Previous animal and human studies have shown that high TGF-β 1 producers develop significantly more lung fibrosis in response to a number of inflammatory triggers such as radiation, 10 chemotherapy, 11 and lung transplantation. (bmj.com)
  • There are no European suggestions on issues explicitly connected with lung transplantation (LTX) in Cystic Fibrosis (CF). The primary objective of this paper is to furnish CF care colleagues with clinically significant CF-explicit data on all parts of LTX, featuring areas of agreement and contention all through Europe. (alliedacademies.org)
  • This audit utilizes explicit proof in the field of lung transplantation in CF patients and resolves all issues of pragmatic significance. (alliedacademies.org)
Burkholderia4
  • Colony morphologies of 96 Burkholderia cenocepacia isolates from cystic fibrosis patients. (drexel.edu)
  • Among these pathogens is Burkholderia cenocepacia - one of the most common and life-threating species found in CF patients that is also easily transmitted and often resistant to antibiotics. (drexel.edu)
  • Burkholderia cepacia complex ( Bcc ) results in worsened clinical course in cystic fibrosis patients. (medscape.com)
  • Identification of Burkholderia and Uncommon Glucose Non-fermenting Gram-Negative Bacilli Isolated from Patients with Cystic Fibrosis using Matrix-Assisted Laser Desorption Ionization-Time of Flight Mass Spectrometry (MALDI-TOF MS). Plongla Rongpong et al. (cdc.gov)
Risk Factors3
  • Understanding the specific risk factors that affect these patients will help clinicians effectively manage their caries risk. (dimensionsofdentalhygiene.com)
  • Symptoms may include fatigue, salty-tasting skin, persistent cough with phlegm, wheezing and shortness of breath, lung infections, and poor growth and weight loss (Cystic Fibrosis Symptoms, Causes & Risk Factors, 2018). (bartleby.com)
  • To assess risk factors for acquisition of P cepacia, 18 cases, defined as any patient with cystic fibrosis with first documented isolation of P cepacia in 1988 or 1989, were compared with 18 matched P cepacia-negative controls with cystic fibrosis. (jasonandjarvis.com)
Mucus and sweat1
30,0001
Clinical trial3
  • Adaptive Phage Therapeutics (APT) recently announced that the first patient in an early-stage clinical trial for its investigative bacteriophage therapy has been dosed. (umn.edu)
  • ic" or the " Corporation") today reported that it had been approved to commence the clinical trial of its orally active anti-fibrotic lead drug candidate, PBI-4050, in patients suffering from cystic fibrosis ("CF"), following the CTA clearance by Health Canada. (newswire.ca)
  • As an active center for multiple clinical trials in CF, Johns Hopkins All Children's was a participating center in a clinical trial program run by Vertex Pharmaceuticals and overseen by the FDA that tested multiple combination therapies to find a therapy to be effective for the majority of patients. (hopkinsmedicine.org)
Adult5
  • A study was undertaken to determine whether B cepacia could be recovered from room air occupied by colonised adult patients with cystic fibrosis. (whiterose.ac.uk)
  • I serve on the Board of Cystic Fibrosis Research, Inc (CFRI) and I am also on the CFRI CF Adult Advisory Committee. (stanford.edu)
  • I joined the Cystic Fibrosis PFAC to educate the Council and the CF adult community about the double lung transplant journey and to work together to make our experience at the Cystic Fibrosis Clinic and Lung Transplnat Clinic at Stanford Hospital a positive, emotional and physical environment for Cystic Fibrosis patients. (stanford.edu)
  • Any interested adult CF patients, CF caregivers, researchers or healthcare providers can be included in the online patient-engagement training program. (who.int)
  • Molecular Epidemiologic Investigation of Mycobacterium intracellulare subspecies chimaera Lung Infections at an Adult Cystic Fibrosis Program. (cdc.gov)
Pathogen2
Hepatic1
  • Cirrhosis Cirrhosis is a late stage of hepatic fibrosis that has resulted in widespread distortion of normal hepatic architecture. (msdmanuals.com)
Antibiotic1
  • For patient one, Jorth and his colleagues surveyed the functional differences between isolates from various lung regions, finding that subpopulations living in upper and lower lobes differed in their growth capacity, stress and antibiotic tolerance, and virulence. (genomeweb.com)
Pediatric4
  • A key to helping patients live even that long -- a vast improvement from an average lifespan of 10 years just decades ago -- is judicious use of antibiotics, explains Andrea Hahn, M.D., a pediatric infectious diseases specialist at Children's National Health System. (mecfa.org)
  • Our CHOC pediatric pulmonologists are board certified in both pediatrics and pediatric pulmonology with additional training in treating cystic fibrosis. (choc.org)
  • A large pediatric cystic fibrosis center. (jasonandjarvis.com)
  • Whole-Genome Sequencing and Epidemiological Analysis Do Not Provide Evidence for Cross-transmission of Mycobacterium abscessus in a Cohort of Pediatric Cystic Fibrosis Patients. (cdc.gov)
Progression2
  • Further work is required to determine whether inhibition of TGF-β 1 activity in these patients may slow disease progression. (bmj.com)
  • This study has several limitations including a small sample size, retrospective single-center design, and a control group that consisted of chronically infected patients who might have a different course of disease progression. (medscape.com)
Exacerbations1
  • Denver - Researchers at National Jewish Health and around the world report in the New England Journal of Medicine that a novel combination of medications improves lung function and reduces disease exacerbations in cystic fibrosis patients with the most common form of the disease. (nationaljewish.org)
Adherence1
  • Semi-directive interviews were realized with three adolescents with cystic fibrosis to understand theirs perceptions about puberty, and their future planning and treatment adherence. (bvsalud.org)
Foundation9
  • 7 the Cystic Fibrosis Foundation, Bethesda, MD. (nih.gov)
  • On Monday, April 1, the CF Foundation joined 16 other patient and consumer groups in filing an amicus brief supporting vital patient protections and coverage expansion included in the Affordable Care Act in the appeal of the Texas v. United States decision, which struck down the constitutionality of the health care law in 2018. (cff.org)
  • Yesterday, the Cystic Fibrosis Foundation joined 16 patient and consumer groups in filing an amicus curiae brief (or friend-of-the-court) in the United States Court of Appeals for the Fifth Circuit case Texas v. United States . (cff.org)
  • The Johns Hopkins All Children's program is nationally accredited by the Cystic Fibrosis Foundation as a Cystic Fibrosis Care Center. (hopkinsmedicine.org)
  • The program is also a Cystic Fibrosis Foundation-designated Therapeutic Development Center and specializes in clinical trials to evaluate the safety and efficacy of new therapies. (hopkinsmedicine.org)
  • Michael Boyle, M.D. , is an internationally recognized cystic fibrosis expert with Johns Hopkins Medicine in Baltimore and senior vice president of therapeutics development of the Cystic Fibrosis Foundation. (hopkinsmedicine.org)
  • Learn about the Cystic Fibrosis Foundation recommendation for checking blood sugar and what CHOP is doing to encourage families to follow these guidelines. (chop.edu)
  • Our Center is selected to be a member of the CF Foundation Therapeutics Development Network (TDN) which is the largest cystic fibrosis clinical trials network in the world. (choc.org)
  • The research was funded by the U.S. National Institutes of Health, the Cystic Fibrosis Foundation and the U.S. Department of Energy. (bnl.gov)
Staphylococcus1
Therapies1
  • In analyzing the results of the SOLUTION study, patients' ability to increase their doses during the study were hindered by time restrictions and amounts allowed per protocol, while other patients were prevented from increasing their dose due to the daily limit (10,000 lipase units/kg/day) for porcine pancreatic enzyme replacement therapies (PERTs). (einpresswire.com)
Treatment7
  • ProMetic has recently reported a statistically and clinically significant decrease in HbA1C levels that compares very favorably to drugs already approved and marketed for the treatment of diabetes as well as additional evidence of PBI-4050's pharmacological and clinical activity in humans based on the analysis of new pro-inflammatory biomarkers in blood and urine samples from these patients. (newswire.ca)
  • At the CHOC Cystic Fibrosis Center, we specialize in providing the very best cystic fibrosis (CF) treatment and care to ensure your child lives a long, healthy life. (choc.org)
  • CHOC has a CF family advisory council open to all families that is designed to improve family-centered care and treatment to our CF patients. (choc.org)
  • Without treatment, most patients die in their 30s. (biotechprimer.com)
  • AzurRx BioPharma shares traded 45% higher after the firm advised it had dosed the first two patients in its Phase 2b OPTION 2 Extension Study of MS1819 for treatment of exocrine pancreatic insufficiency in patients with cystic fibrosis. (streetwisereports.com)
  • The company relayed that the first two patients have now been dosed at higher dosage levels and will continue treatment for a total of two weeks. (streetwisereports.com)
  • Vertex's first licensed treatment, Kalydeco, can be used for around 8% of patients. (politicshome.com)
Isolates1
  • After sequencing the genomes of 96 isolates from the upper, middle, and lower lobes from three patients, the researchers constructed phylogenetic trees. (genomeweb.com)
Molecular1
  • Recent studies that applied molecular approaches to identify unusual pathogens in patients with CF showed various infrequently encountered and novel species ( 4 - 7 ). (cdc.gov)
Pancreatic3
  • The objectives of this 24 week randomized, double-blind, and placebo-controlled Phase 2 study includes the evaluation of the effects of PBI-4050 on pancreatic and lung function in 90 CF patients. (newswire.ca)
  • Approximately 85-90% of patients with cystic fibrosis (CF) suffer from pancreatic insufficiency (PI), which predisposes them to malabsorption of lipids and lipid soluble vitamins leading to a deficiency of nonenzymatic antioxidants [ 2 ]. (hindawi.com)
  • HAYWARD, Calif., Dec. 27, 2016 (GLOBE NEWSWIRE) -- Anthera Pharmaceuticals, Inc. (NASDAQ:ANTH) today announced the top line results of the SOLUTION clinical study in cystic fibrosis patients with exocrine pancreatic insufficiency (EPI). (einpresswire.com)
Sweat2