VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes)

Condition:   Cystic Fibrosis Interventions:   Drug: ELX/TEZ/IVA;   Drug: IVA Sponsor:   Vertex Pharmaceuticals Incorporated Available
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials

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Publication date: Available online 13 September 2019Source: Journal of Ginseng ResearchAuthor(s): Do-Yeon Cho, Daniel Skinner, Shaoyan Zhang, Ahmed Lazrak, Dong Jin Lim, Christopher G. Weeks, Catherine G. Banks, Chang Kyun Han, Si-Kwan Kim, Guillermo J. Tearney, Sadis Matalon, Steven M. Rowe, Bradford A. WoodworthAbstractBackgroundAbnormal chloride (Cl−) transport has a detrimental impact on mucociliary clearance in both cystic fibrosis (CF) and non-CF chronic rhinosinusitis (CRS). Ginseng is a medicinal plant noted to have anti-inflammatory and antimicrobial properties. The present study aims to assess the capabilit...
Source: Journal of Ginseng Research - Category: Complementary Medicine Source Type: research
ConclusionViral infections contributed to the increase in morbidity and mortality of CF patients in our population, and rhinovirus was the most common causative agent. Viral infections and viral recurrence increased the prevalence of bacterial infection of specific pathogens such as H. influenza and S. aureus. Physicians should be aware to prevent progressive lung damage in CF patients by treating the concomitant viral and bacterial infections. Viral infection may be associated with some common CFTR mutations.
Source: International Journal of Pediatrics and Adolescent Medicine - Category: Pediatrics Source Type: research
Children with CF have been reported to consume significantly more energy-dense, nutrient-poor foods than controls where there are now concerns of inadequate micronutrient intake. There are no current or comprehensive dietary studies assessing micronutrient intake in CF children.
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Source Type: research
A five-year deal to supply NHS Scotland with cystic fibrosis drugs Orkambi and Symkevi has been secured after discussions between the manufacturer, Vertex Pharmaceuticals, and the Scottish government.
Source: The Pharmaceutical Journal - Category: Drugs & Pharmacology Source Type: research
Pharmaceutical bosses have been summoned to a meeting with Health Secretary Matt Hancock in an attempt to break a four-year deadlock over the cost of cystic fibrosis drugs.
Source: the Mail online | Health - Category: Consumer Health News Source Type: news
Source: BMJ News - Category: General Medicine Source Type: research
Amid Vertex ’s continuing drug pricing battle with British regulators, the drugmaker has signed a deal to make two of its cystic fibrosis drugs available in Scotland.
Source: PharmaManufacturing.com - Category: Pharmaceuticals Source Type: news
Publication date: Available online 11 September 2019Source: Sensors and Actuators B: ChemicalAuthor(s): Pasqualina D’Ursi, Matteo Uggeri, Chiara Urbinati, Enrico Millo, Giulia Paiardi, Luciano Milanesi, Robert C. Ford, Jack Clews, Xin Meng, Paolo Bergese, Andrea Ridolfi, Nicoletta Pedemonte, Paola Fossa, Alessandro Orro, Marco RusnatiAbstractCystic fibrosis (CF) is mainly caused by the mutation F508del of the cystic fibrosis transmembrane conductance regulator (CFTR) that is thus retained in the endoplasmic reticulum and degraded. New drugs able to rescue F508del-CFTR trafficking and activity are eagerly awaited, a g...
Source: Sensors and Actuators B: Chemical - Category: Chemistry Source Type: research
Definition of Pseudomonas aeruginosa (Pa) microbiological status is essential for patients' inclusion in clinical trials. The aim of this study was to agree on the definitions of Pa infection status for initial infection, eradication and chronic infection to be used in clinical trials and to propose additional future study areas.
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Original Article Source Type: research
Conclusion: Good2Go is a valid 20-item questionnaire to assess transition readiness in adolescents with chronic conditions and may be useful in routine care to propose individually tailored preparation for their transfer to adult healthcare. Further research is now needed to analyze correlation between domain scores and success of transition.What is Known:•In adolescents with chronic conditions, the use of transition readiness questionnaires is recommended to propose individually tailored preparation for their transfer to adult healthcare.•However, no French-language questionnaire has been so far validated.What i...
Source: European Journal of Pediatrics - Category: Pediatrics Source Type: research
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