Cystic fibrosis patients offered 'life-transforming' drug

The three-drug combination, Kaftrio, will now be available on the NHS in England.
Source: BBC News | Health | UK Edition - Category: Consumer Health News Source Type: news

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Cancers, Vol. 12, Pages 1801: Role of Curcumin and (−)-Epigallocatechin-3-O-Gallate in Bladder Cancer Treatment: A Review Cancers doi: 10.3390/cancers12071801 Authors: Ludwika Piwowarczyk Maciej Stawny Dariusz T. Mlynarczyk Izabela Muszalska-Kolos Tomasz Goslinski Anna Jelińska The incidence of bladder cancer (BC) is increasing, and although current therapeutic approaches are effective in many cases, recurrence of BC is common. Therefore, it seems necessary to search not only for novel therapeutic approaches, but also for new therapeutic agents. Natural polyphenols, such as curcumin (CUR) and epiga...
Source: Cancers - Category: Cancer & Oncology Authors: Tags: Review Source Type: research
Over the past six months, the MoS has lifted the lid on the secretive talks between NHS England and drug company Vertex on access to cystic fibrosis drugs.
Source: the Mail online | Health - Category: Consumer Health News Source Type: news
Fibrosis occurs in most human organs including the liver, lung, heart and kidney, and is crucial for the progression of most chronic diseases. As an indispensable catabolic process for intracellular quality control and homeostasis, autophagy occurs in most mammalian cells and is implicated in many biological processes including fibrogenesis. Although advances have been made in understanding autophagy process, the potential role of autophagy in fibrotic diseases remains controversial and has recently attracted a great deal of attention. In the current review, we summarize the commonalities of autophagy affecting different t...
Source: Theranostics - Category: Molecular Biology Authors: Tags: Review Source Type: research
Many proteobacteria, such as Escherichia coli, contain two main types of quinones: benzoquinones, represented by ubiquinone (UQ) and naphthoquinones, such as menaquinone (MK), and dimethyl-menaquinone (DMK). MK and DMK function predominantly in anaerobic respiratory chains, whereas UQ is the major electron carrier in the reduction of dioxygen. However, this division of labor is probably not very strict. Indeed, a pathway that produces UQ under anaerobic conditions in an UbiU-, UbiV-, and UbiT-dependent manner has been discovered recently in E. coli. Its physiological relevance is not yet understood, because MK and DMK are ...
Source: Journal of Biological Chemistry - Category: Chemistry Authors: Tags: Metabolism Source Type: research
MO Abstract With its high detail of morphological changes in lung parenchyma and airways as well as the possibilities for three-dimensional reconstruction, computed tomography (CT) represents a solid tool for the diagnosis and follow-up in patients suffering from cystic fibrosis (CF). Guidelines for standardized CT image acquisition in CF patients are still missing. In the mostly younger CF patients, an important issue is the well-considered use of radiation in CT imaging. The use of intravenous contrast agent is mainly restricted to acute emergency diagnostics. Typical morphological findings in CF lung dise...
Source: Der Radiologe - Category: Radiology Authors: Tags: Radiologe Source Type: research
CONCLUSIONS: Reports on the use of hematopoietic stem cell transplantation improving survival and preventing symptoms and complications associated with sickle cell disease are currently limited to observational and other less robust studies. We did not find any eligible randomized controlled trials assessing the benefit or risk of hematopoietic stem cell transplantations. However, there is an ongoing quasi-randomized trial comparing hematopoietic stem cell transplantation with standard care, Thus, this systematic review identifies the need for a multicentre randomized controlled trial assessing the benefits and possible ri...
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
AireHealth, a medtech company based in Orlando, Florida, currently offers a portable nebulizer and companion app for respiratory patients aged two and over. The small nebulizer can be charged using a micro USB charger and then placed in a bag or pock...
Source: Medgadget - Category: Medical Devices Authors: Tags: Exclusive Informatics Medicine Telemedicine AireHealth Source Type: blogs
We present 4 paediatric cases to raise the awareness of electrolyte disturbances in CF patients. PMID: 32603052 [PubMed - in process]
Source: Acta Gastro-Enterologica Belgica - Category: Gastroenterology Tags: Acta Gastroenterol Belg Source Type: research
CONCLUSIONS: There is insufficient evidence from randomised trials to determine whether conservative preoperative blood transfusion is as effective as aggressive preoperative blood transfusion in preventing sickle-related or surgery-related complications in people with HbSS disease. There is very low quality evidence that preoperative blood transfusion may prevent development of acute chest syndrome. Due to lack of evidence this review cannot comment on management for people with HbSC or HbSβ+ disease or for those with high baseline haemoglobin concentrations. PMID: 32614473 [PubMed - as supplied by publisher]
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
Nontuberculous mycobacteria (NTM) are environmental organisms found in soil and water worldwide that can cause chronic lung infection, usually in the context of lung disease or immunodeficiency. Cystic fibrosis (CF) has been identified as the disease population at greatest risk for CF airway infection [1]. Within the CF population, the vast majority of infections occur as a result of either species within the M. avium complex (MAC) or subspecies of M. abscessus (MABC). In the largest longitudinal survey to date, 20% of people with CF who had NTM cultures obtained over a 5-year interval had a positive culture for NTM [2].
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Source Type: research
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