Cystic Fibrosis drugs scandal: Hope as NHS tables new offer for life-changing drug

A NEW, improved NHS offer for life-saving drugs was welcomed by cystic fibrosis sufferers last night. The Daily Express has been pressurising NHS England to strike a deal with US drugs giant Vertex for its pipeline of CF medicines like Orkambi.
Source: Daily Express - Health - Category: Consumer Health News Source Type: news

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CONCLUSION: After more than a decade, lung MRI has become a valuable tool for monitoring CF in clinical routine application and as an endpoint for clinical studies. PMID: 31172247 [PubMed - as supplied by publisher]
Source: Der Radiologe - Category: Radiology Authors: Tags: Radiologe Source Type: research
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Posters Sessions Source Type: research
The objective of the study was to develop a method to visualize and quantify MCC in pulmonary pathophysiology using PET imaging. Pre-clinical imaging was performed using a porcine model. Methods: [18F]-fluoride labeled alumina nanoparticles were obtained by adding aqueous [18F]-fluoride to -alumina nanoparticles (
Source: Journal of Nuclear Medicine - Category: Nuclear Medicine Authors: Tags: Outcomes/Infectious Disease/Pulmonary II Source Type: research
A TODDLER with cystic fibrosis will deliver a bunch of 65 roses to Prime Minister Theresa May today, to plead for life-saving drugs to be available on the NHS. Eve Jones, one, was born with the cruel, deteriorating genetic condition, but wonder drug Orkambi could add decades to her life.
Source: Daily Express - Health - Category: Consumer Health News Source Type: news
Abstract Asthma has been implicated as a driving force in lower airway remodeling; however, its effect on upper airway development has not been studied. Clinical disease, particularly cystic fibrosis (CF), has been associated with anatomical paranasal sinus variation, although the mechanism for these variations remains unclear. The purpose of this study was to determine whether asthma is associated with altered sinus pneumatization. Five hundred ninety-one computed tomography scans, including 303 adolescents (age 13-18) and 288 adults (age> 18), were evaluated using the Assessment of Pneumatization of the Paran...
Source: Ear, Nose and Throat Journal - Category: ENT & OMF Authors: Tags: Ear Nose Throat J Source Type: research
Elvira Sondo1, Roberta Bertelli2, Emanuela Pesce1, Gian Marco Ghiggeri2 and Nicoletta Pedemonte1* 1UOC Genetica Medica, IRCCS Istituto Giannina Gaslini, Genova, Italy 2UOC Nefrologia, IRCCS Istituto Giannina Gaslini, Genova, Italy Neutrophils migrate to sites of infection where they phagocytose, degranulate, and/or, in the presence of appropriate stimuli, release decondensed chromatin strands (called neutrophil extracellular traps, NETs) for trapping and possibly killing microorganisms. NET formation is characterized by marked morphological cell changes, in particular within the nucleus. Lytic NET formation can ...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Cosmeri Rizzato1, Javier Torres2, Elena Kasamatsu3, Margarita Camorlinga-Ponce2, Maria Mercedes Bravo4, Federico Canzian5 and Ikuko Kato6* 1Department of Translation Research and of New Technologies in Medicine and Surgery, University of Pisa, Pisa, Italy 2Unidad de Investigación en Enfermedades Infecciosas, Unidades Médicas de Alta Especialidad Pediatría, Instituto Mexicano del Seguro Social, Mexico City, Mexico 3Instituto de Investigaciones en Ciencias de la Salud, National University of Asunción, Asunción, Paraguay 4Grupo de Investigación en Biología del C&aacut...
Source: Frontiers in Microbiology - Category: Microbiology Source Type: research
Mark E. Gray1,2*, James Meehan2,3, Paul Sullivan4, Jamie R. K. Marland4, Stephen N. Greenhalgh1, Rachael Gregson1, Richard Eddie Clutton1, Carol Ward2, Chris Cousens5, David J. Griffiths5, Alan Murray4 and David Argyle1 1The Royal (Dick) School of Veterinary Studies and Roslin Institute, University of Edinburgh, Edinburgh, United Kingdom 2Cancer Research UK Edinburgh Centre and Division of Pathology Laboratories, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, United Kingdom 3School of Engineering and Physical Sciences, Institute of Sensors, Signals and Systems, Heriot-Watt Univer...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
Conclusion To conclude, this study suggests a preserved plasticity in the skeletal muscle of a patient with MELAS. More importantly, Resistance Training appears to be a safe and effective method to increase skeletal muscle function in this patient population, and this effect is mediated by both neuromuscular and mitochondrial adaptations. However, particular attention and caution is needed in the interpretation of the data of this single case study and further studies are warranted including larger sample of patients. Ethics Statement For this case study the participant caregiver provided written informed consent. Auth...
Source: Frontiers in Physiology - Category: Physiology Source Type: research
Jennifer M. Rudd1, Sivasami Pulavendran1, Harshini K. Ashar1, Jerry W. Ritchey1, Timothy A. Snider1, Jerry R. Malayer1, Montelongo Marie1, Vincent T. K. Chow2 and Teluguakula Narasaraju1* 1Center for Veterinary Health Sciences, Oklahoma State University, Stillwater, OK, United States2Department of Microbiology and Immunology, Yong Loo Lin School of Medicine, National University of Singapore, National University Health System, Singapore, Singapore Exaggerated host innate immune responses have been implicated in severe influenza pneumonia. We have previously demonstrated that excessive neutrophils recruited during in...
Source: Frontiers in cellular and infection microbiology - Category: Microbiology Source Type: research
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