New Treatment for Cystic Fibrosis

Trikafta, a combination of elexacaftor, ivacaftor, and tezacaftor, is a new drug for the treatment of the most common cystic fibrosis mutation.Serious adverse effects include elevated liver enzymes and an increased risk of cataracts.
Source: AJN - Category: Nursing Tags: Drug Watch Source Type: research

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Conclusion: Tocilizumab is an effective therapeutic option for reducing disease activity in children with refractory non-anterior uveitis. PMID: 32058831 [PubMed - as supplied by publisher]
Source: Ocular Immunology and Inflammation - Category: Allergy & Immunology Tags: Ocul Immunol Inflamm Source Type: research
Conclusion: The clinically approved NKR-1 receptor antagonist Fosaprepitant decreases NKR-1 protein expression effectively not only in the exposed but also in the unexposed partner eye in a UVR-B irradiation mouse model. No effect was seen on the protein concentration of pro-inflammatory cytokines/chemokines in either eye. PMID: 32058829 [PubMed - as supplied by publisher]
Source: Ocular Immunology and Inflammation - Category: Allergy & Immunology Tags: Ocul Immunol Inflamm Source Type: research
Authors: Cheng PC, Alexiou S, Rubenstein RC Abstract INTRODUCTION: Cystic fibrosis (CF) is the most common autosomal recessive disorder affecting approximately 70,000 people worldwide. The lack of functional cystic fibrosis transmembrane conductance regulator (CFTR) causes dysregulation of epithelial fluid transport in the lungs, gastrointestinal tract, and sweat glands. Areas covered: The most common disease-causing CFTR mutation, F508del, is present in nearly 75% of those affected and results in a defective protein. Therapies to improve the function of this mutant protein have the promise to reduce morbidity and ...
Source: Expert Review of Respiratory Medicine - Category: Respiratory Medicine Tags: Expert Rev Respir Med Source Type: research
CONCLUSIONS: We were unable to identify any eligible studies for inclusion in this review and hence it is not possible to draw any conclusions based on randomised controlled studies. However, we are aware of uncontrolled studies which support the efficacy of newborn screening for galactosaemia. There are a number of reviews and economic analyses of non-trial literature suggesting that screening is appropriate. PMID: 29274129 [PubMed - as supplied by publisher]
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
Introduction: Transition from specialist paediatric to adult services is a crucial time &while this process is well established in other chronic conditions (e.g. Cystic Fibrosis) there is a lack of information in an asthma setting.Aims &Objectives: We were interested in the features of young adults (YAs) transitioning from paediatric to adult asthma services.Methods: We retrospectively reviewed our referrals from the paediatric sector over the last 4years.Results: All 42 referrals [40.5% female] transitioned between the ages of 16-18yrs. Almost half the males were below the 25th centile for height at the time of tr...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Paediatric Asthma and Allergy Source Type: research
This article reviews safety of this therapy. Areas covered: Safety findings in ivacaftor, lumacaftor and combined therapy trials, and reported subsequently through post-approval evaluation, were accessed by PubMed and Google searches using key words "VX-770", "ivacaftor", "VX-809", and "lumacaftor". Transaminitis was seen in ivacaftor and combination trials. Non-congenital cataracts were seen in pre-clinical animal studies and in children taking ivacaftor and combined therapy. Dyspnea occurs in some patients taking lumacaftor and combined therapy and usually resolves without stopping...
Source: Expert Opinion on Drug Safety - Category: Drugs & Pharmacology Tags: Expert Opin Drug Saf Source Type: research
Research and progress on ClC‑2 (Review). Mol Med Rep. 2017 May 18;: Authors: Wang H, Xu M, Kong Q, Sun P, Yan F, Tian W, Wang X Abstract Chloride channel 2 (ClC-2) is one of the nine mammalian members of the ClC family. The present review discusses the molecular properties of ClC‑2, including CLCN2, ClC‑2 promoter and the structural properties of ClC‑2 protein; physiological properties; functional properties, including the regulation of cell volume. The effects of ClC‑2 on the digestive, respiratory, circulatory, nervous and optical systems are also discussed, in addition to the mechanisms in...
Source: Molecular Medicine Reports - Category: Molecular Biology Tags: Mol Med Rep Source Type: research
CONCLUSIONS: The study suggests that the risk of developing cataract was higher among patients with CF than among the GP. PMID: 28394650 [PubMed - as supplied by publisher]
Source: Ophthalmic Genetics - Category: Opthalmology Tags: Ophthalmic Genet Source Type: research
This article reviews ivacaftor safety. Areas covered: Safety findings in ivacaftor clinical trials, and reported subsequently, were accessed by a PubMed search using key words "VX-770" or "ivacaftor". Additional information was accessed via Google Search. Transaminitis was noted in ivacaftor and combination lumacaftor-ivacaftor trials. Ivacaftor was associated with cataracts in juvenile rat pups in pre-clinical studies; non-congenital cataracts have been found in children taking ivacaftor. Ivacaftor is a CYP3A substrate; CYP3A inhibitors and inducers should be avoided during its administration. Ivacafto...
Source: Expert Opinion on Drug Safety - Category: Drugs & Pharmacology Tags: Expert Opin Drug Saf Source Type: research
I have been on this site a few years ago and have decided to return because I am in great need of help, guidance and advice. MY SITUATIONS: I am a 29 year old female from California. I have cystic fibrosis, cystic fibrosis related type one diabetes, I had a double lung transplant 5 years ago and now have numerous transplant complications including 2 types of aggressive skin cancer, cataracts (now currently being treated), chronic pain, osteoarthritis and chronic pain. I have a college degree in anthropology, work as a freelance writer and in customer service and work as a volunteer for several different organizations. T...
Source: Cystic Fibrosis Adults Forum - Category: Respiratory Medicine Authors: Tags: Adults Source Type: forums
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