Gene therapy breakthrough for cystic fibrosis

ConclusionThis RCT showed that a new non-viral-based gene therapy for cystic fibrosis was able to produce “modest” benefits in lung function compared to a placebo. The treatments were given once a month for a year. The study had many strengths, including its double-blind randomised design, recruiting adequate numbers to demonstrate real differences between groups, and using pre-specified outcomes and sub-analysis. This means we can be confident in the reliability of the findings presented. Although the findings of this study are encouraging, there are always limitations. These include: This study was relatively small, recruiting just 140 patients. This is normal for a phase II trial, but large clinical trials are needed to fully assess the effects and safety of this treatment in development. Patients recruited in this trial had to be clinically stable to be included. This means they might be at their optimum respiratory health at this stage. Therefore, we don’t know how the treatment would work in clinically unstable or very severe patient groups.It is important to realise that both groups’ lung function got worse over the year, so the treatment as it stands is quite limited. The new gene therapy was able to lessen some of the deterioration, but not in all. Nonetheless, this gives the researchers hope and scope to work out how to improve it. Optimising the dose, working out why it worked in some people and not others, and trialling the therapy in mor...
Source: NHS News Feed - Category: Consumer Health News Tags: Genetics/stem cells Heart/lungs Source Type: news

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This study is registered with, number NCT02725567. Findings Children aged 12 to <24 months were enrolled between Aug 25, 2016, and Nov 1, 2017. Seven children were enrolled in part A, of whom five received 50 mg and two received 75 mg ivacaftor. All completed treatment. Of 19 children enrolled in part B, including one from part A, all received 50 mg ivacaftor and 18 completed treatment (one withdrew because of difficulty with blood draws). All children received at least one dose of ivacaftor. Pharmacokinetics indicated exposure was similar to that in children aged 2 to <6 years and adults. No child...
Source: The Lancet Respiratory Medicine - Category: Respiratory Medicine Source Type: research
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Poster Sessions Source Type: research
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Poster Sessions Source Type: research
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Workshops Source Type: research
This study is registered with the UK Clinical Research Network (14615) and with the International Standard Randomised Controlled Trial Network Registry (12473810). Findings Between Jan 23, 2012, and July 4, 2017, 124 patients were prospectively recruited to the trial and had 200 sputum induction procedures for stage 1. 167 (84%) procedures were successful and the procedure was well tolerated. Of the 167 paired samples, 63 (38%) sputum-induction samples were pathogen positive compared with 24 (14%) cough swabs (p<0·0001; odds ratio [OR] 7·5; 95% CI 3·19–17·98). More pathogens were isolat...
Source: The Lancet Respiratory Medicine - Category: Respiratory Medicine Source Type: research
Publication date: Available online 16 May 2018 Source:The Lancet Respiratory Medicine Author(s): André Schultz, Daan Caudri
Source: The Lancet Respiratory Medicine - Category: Respiratory Medicine Source Type: research
People with cystic fibrosis, muscular dystrophy, and other chronic conditions can have serious difficulty coughing. This creates not only discomfort, but potentially dangerous complications for the lungs and heart. There are cough assist devices on t...
Source: Medgadget - Category: Medical Devices Authors: Tags: Medicine Pediatrics Rehab Thoracic Surgery Source Type: blogs
Discussion Normal kidneys regulate water balance to maintain a plasma osmolality of 275-290 mOsm/kg normally. Thirst and arginine vasopressin or antidiuretic hormone (ADH) are the primary regulators of plasma osmolality. ADH is made in the hypothalamus and released by the posterior pituitary gland. ADH acts on the kidney’s distal collecting duct to increase water reabsorption. ADH is appropriately released in hypovolemic states, such as dehydration caused by gastroenteritis. ADH has an ~10 minute half-life and therefore can respond to rapid changes in volume status. Sodium balance is regulated by aldosterone (as part...
Source: - Category: Pediatrics Authors: Tags: Uncategorized Source Type: news
CONCLUSIONS: Diagnosis of non-CF bronchiectasis is often delayed because of a failure to recognize the significance of symptoms. Through clinical investigation, including a HRCT scan of the chest, sweat test, studies of immune function, and ciliary function in a child with a prolonged suppurative cough, remains important. In Turkey, the most common causes of non-CF bronchiectasis are PCD and immunodeficiency, related to a high frequency of consanguinity. PMID: 29605210 [PubMed - in process]
Source: Respiratory Care - Category: Respiratory Medicine Authors: Tags: Respir Med Source Type: research
CONCLUSIONS: There is currently no evidence indicating whether orally administered antibiotics are more beneficial compared to inhaled antibiotics. The recent ERS bronchiectasis guidelines provide a practical approach to the use of long-term antibiotics. New research is needed comparing inhaled versus oral antibiotic therapies for bronchiectasis patients with a history of frequent exacerbations, to establish which approach is the most effective in terms of exacerbation prevention, quality of life, treatment burden, and antibiotic resistance. PMID: 29587336 [PubMed - as supplied by publisher]
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
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