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Airway Abnormality, Immunodeficiency Are Key Causes of Non-CF Bronchiectasis in Turkey, Study Finds

The most common causes of non-cystic fibrosis bronchiectasis in Turkish children are an airway abnormality called primary ciliary dyskinesia and a sub-par immune system, a study reports. A high frequency of marriages between relatives may be related to the conditions, researchers said. The study, “A review of the etiology and clinical presentation of non-cystic fibrosis bronchiectasis: A tertiary care experience,” was published in the journal Respiratory Medicine. Although cystic fibrosis is the most common cause of bronchiectasis, it can have other causes. These include infections, pneumonia, lung problems, congenital problems, immune deficiency, and breathing toxic gases or particles. In r

Eluforsen for cystic fibrosis

ProQR is developing an investigational product, eluforsen (QR-010) for people with cystic fibrosis (CF) that have the F508del mutation. F508del is the most common mutation in CF and affects more than 85% of all CF patients. Despite significant efforts in drug development, people with CF still have a limited life expectancy. Eluforsen is being developed as a regularly inhaled therapy. Unlike any other CF drug currently on the market, it is designed to bind specifically to the defective CFTR RNA and to restore the function of the CFTR protein. With this we aim to treat the underlying cause of the disease and stop the progression of cystic fibrosis. Two global clinical trials for eluforse

Staphylococcus aureus in Patients Diagnosed With Bronchiectasis Without Cystic Fibrosis

Patients diagnosed with bronchiectasis and in whom sputum cultures grow Staphylococcus aureus may not experience a difference in exacerbation frequency, forced expiratory volume in 1 second (FEV1) decline, or hospital admission rates compared with patients with bronchiectasis in whom sputum cultures do not grow S aureus after adjusting for patient characteristics, according to research published in the Annals of the American Thoracic Society. Using the United States Bronchiectasis Research Registry, investigators identified 830 patients diagnosed with bronchiectasis from 13 sites. Individuals were placed into 1 of 3 groups based upon the organism that grew during previous respiratory culture

Early Growth Patterns Affect CF Children’s Lung Function at Age 12, Study Shows

Children with cystic fibrosis who are able to recover from below-normal growth in their first two years are more likely to achieve normal growth and better lung condition by 12 years of age, a study shows. The research, “Early life growth patterns persist for 12 years and impact pulmonary outcomes in cystic fibrosis,” was published in the Journal of Cystic Fibrosis. Scientists have long known there is a link between young CF patients’ ability to respond to nutrition and their lung condition. The clinical trials Responder I, II and III have been exploring the benefits that children with CF attain when they respond well to nutrition. Responder I defined responders as those whose weight recover

Vertex’s VX-659 Triple Combo To Be Tested in Phase 3 Trial in Patients with 2 Copies of F508del Muta

Vertex Pharmaceuticals is launching a new Phase 3 trial to assess the triple combination of VX-659 plus tezacaftor (VX-661) and Kalydeco (ivacaftor) in cystic fibrosis (CF) patients with two copies of the F508del mutation in the CFTR gene. That mutation most common CF-causing genetic variant. The trial will enroll about 100 patients, ages 12 or older. Participants will be randomized to receive placebo or the oral triple combination — 240 mg of VfX-659 plus 100 mg tezacaftor and 150 mg Kalydeco each morning, and a 150 mg Kalydeco table again at night — for four weeks. All patients will be given tezacaftor and Kalydeco in combination for four weeks prior to the start of the treatment period. A

Verona Pharma Reports Positive Top-Line Data from Phase 2a Trial with RPL554 in Cystic Fibrosis Pati

RPL554 demonstrates a favorable pharmacokinetic and pharmacodynamic profile in cystic fibrosis patients LONDON, UK I March 02, 2018 I Verona Pharma plc (AIM:VRP) (Nasdaq:VRNA) (“Verona Pharma”), a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for respiratory diseases, announces today that top-line data from its Phase 2a trial with nebulized RPL554 demonstrate that the investigational therapy has a favorable pharmacokinetic (PK) and pharmacodynamic (PD) profile, with single doses achieving statistically significant (P<0.05) increases in average forced expiratory volume in one second (FEV1) in patients with cystic fibrosis (CF). These d

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