Long-awaited cystic fibrosis drug could turn deadly disease into a manageable condition

Thirty years after scientists discovered the defective gene that causes cystic fibrosis, two new trials show a therapy could help 90 percent of patients. The FDA approved the triple drug, Trikafta, five months ahead of its deadline.
Source: Washington Post: To Your Health - Category: Consumer Health News Authors: Source Type: news

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Authors: Donadio MVF, Vendrusculo FM, Pérez-Ruiz M PMID: 32944304 [PubMed]
Source: Journal of Thoracic Disease - Category: Respiratory Medicine Tags: J Thorac Dis Source Type: research
Conditions:   Salt; Deficit (or Low);   Cystic Fibrosis Interventions:   Diagnostic Test: fractional sodium excretion;   Other: risk subgroups Sponsor:   University Hospital, Ghent Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
No abstract available
Source: Postgraduate Obstetrics and Gynecology - Category: OBGYN Tags: Article Source Type: research
CONCLUSIONS: Our results show that inhibition of protease activity elicits anti-inflammatory effects in CF mice with acute P. aeruginosa lung infection. Thus, Marimastat and Ilomastat represent candidate molecules for the treatment of CF patients, encouraging further studies on protease inhibitors and their application in inflammatory diseases. PMID: 32946591 [PubMed - as supplied by publisher]
Source: Clinical and Developmental Immunology - Category: Allergy & Immunology Authors: Tags: Clin Exp Immunol Source Type: research
Background: Non-cystic fibrosis (CF) bronchiectasis is a significant but often under-recognised cause of respiratory morbidity in children. Current understanding of the risk factors leading to the development, and subsequent exacerbations of non-CF bronchiectasis is limited. Exacerbations requiring hospitalisation are associated with a more rapid decline in lung function. Our study aimed to identify risk factors for severe respiratory exacerbations in children with non-CF bronchiectasis.
Source: International Journal of Infectious Diseases - Category: Infectious Diseases Authors: Tags: 0537 Source Type: research
We examined the pathogen-specific nature of this autoreactivity by examining antibodies to BPI in bacteremia patients. Antibodies to BPI and bacterial antigens were measured in sera by ELISA from five patient cohorts (n = 214). Antibody avidity was investigated. Bacteremic patient sera (n = 32) exhibited IgG antibody autoreactivity against BPI in 64.7% and 46.7% of patients with positive blood cultures for P. aeruginosa and Escherichia coli, respectively. Autoantibody titers correlated with IgG responses to bacterial extracts and lipopolysaccharide (LPS). A prospective cohort of bacteremic patient sera exhibited anti-BPI I...
Source: Infection and Immunity - Category: Infectious Diseases Authors: Tags: Host Response and Inflammation Source Type: research
Authors: Shan W, Hu Y, Ding J, Yang X, Lou J, Du Q, Liao Q, Luo L, Xu J, Xie R Abstract Intracellular calcium (Ca2+) is a critical cell signaling component in gastrointestinal (GI) physiology. Cytosolic calcium ([Ca2+]cyt), as a secondary messenger, controls GI epithelial fluid and ion transport, mucus and neuropeptide secretion, as well as synaptic transmission and motility. The key roles of Ca2+ signaling in other types of secretory cell (including those in the airways and salivary glands) are well known. However, its action in GI epithelial secretion and the underlying molecular mechanisms have remained to be fu...
Source: Experimental and Therapeutic Medicine - Category: General Medicine Tags: Exp Ther Med Source Type: research
Infant pulmonary function testing using the raised volume rapid thoracoabdominal compression (RVRTC) technique requires sedation and is time consuming. Many cystic fibrosis (CF) centers do not have access to equipment and the utility of routine testing remains to be determined. We aimed to assess whether RVRTC tests performed during infancy predict spirometry at early school age.
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Short communication Source Type: research
Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) has disrupted clinical trials worldwide [1]. This could delay the approval of new medicines and reduce access to investigational treatments via clinical trials. This particularly impacts patients with rare diseases such as cystic fibrosis (CF).
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Original Articles: Correspondence Source Type: research
Publication date: Available online 16 September 2020Source: Respiratory Medicine Case ReportsAuthor(s): Alicia A. Mirza, Elika J. Rad, Paul K. Mohabir
Source: Respiratory Medicine Case Reports - Category: Respiratory Medicine Source Type: research
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