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Personalized Nutrition Programs May Help Improve Outcomes for CF Patients, Survey Suggests

New personalized strategies that can address the nutritional needs of cystic fibrosis patients may provide the best care and improve patient outcomes, a study suggests. The study, “A survey identifying nutritional needs in a contemporary adult cystic fibrosis cohort,” was published in the journal BMC Nutrition. CF is mainly known for its progressive respiratory symptoms. However, the underlying disease-causing CFTR gene mutations can also alter the function of the pancreas, causing gastrointestinal symptoms, CF-related diabetes, impaired absorption of nutrients, and other clinically important non-pulmonary manifestations. Malnutrition in the CF population has been considered a critical probl

The Diverse Spectrum of Health Across the CF Community

If you’ve ever met more than one person with cystic fibrosis, it’s likely they had very different experiences than you. First, their approach likely will be different; chronic disease affects everyone differently mentally, so some are vocal and “embrace” it, while others are quieter and choose to consider it a personal journey. Both approaches are fine, it’s just that we act differently. CF also affects everyone differently physically. Over the years, people traditionally have referred to CF as a “lung disease” because that’s the most commonly known clinical manifestation of illness. But for most of those with CF, the lungs are only a part of it, and for some, not even the worst part. CF is

At-home DNA test kits: Medical experts warn buyers beware

(WRIC) - Did you or someone you know get an at-home DNA test kit for Christmas? They were one of the hottest holiday items. It's an easy way to learn about your ethnic background, and now some promise to reveal potential health risks. Medical experts, however, say buyer beware. They tell our sister station 8News there's nothing more private and personal than your genetic information, and if you're not comfortable giving someone your social security number, you need to think twice about sharing your DNA. Medical professionals also warn that there's a difference between the pricey doctor ordered test at a certified lab and spit kits available online. "There are some studies that suggest the er

Non-invasive Ventilation Improves Lung Function in CF Patients But Not Survival, Study Finds

A reassessment of U.K. medical records showed that in cystic fibrosis (CF) patients, breathing support provided by gas inhalation, through a face or nasal mask, helps improve lung function but not survival, researchers report. The study “Non-invasive ventilation and clinical outcomes in cystic fibrosis: Findings from the UK CF registry” was published in the Journal of Cystic Fibrosis. Airway obstruction in CF patients leads to difficulties in breathing, potentially resulting in respiratory failure. Non-invasive ventilation (NIV) therapy — the use of a face or nasal mask to provide inhalation gases for breathing support — is a commonly used method to aid airway clearance, reduce muscle fatigu

Cystic fibrosis drug combo found safe and effective for 2–5 year olds

An open label phase 3 study conducted at Ann & Robert H. Lurie Children’s Hospital of Chicago and other centers established that a combination of cystic fibrosis drugs lumacaftor and ivacaftor is safe and effective in children aged 2–5 years whose disease is caused by two copies of the F508del-CFTR gene mutation—the most common and severe form of cystic fibrosis. These data, published in Lancet Respiratory Medicine, were the basis for the Food and Drug Administration’s extended approval for this treatment to include children 2 years and older. Previously, the drug combination was approved for patients 6 years and older. “With earlier treatment that targets the genetic cause of cystic fibrosi

Eluforsen Improves CFTR Protein Function in CF Patients with F508del Mutations, Phase 1 Trial Shows

An exploratory Phase 1 trial showed that repeated intranasal treatment with the therapy eluforsen improved CFTR protein activity in adults with cystic fibrosis (CF) carrying F508del mutations in both CFTR gene copies. The study, “Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis,” was published in the Journal of Cystic Fibrosis. CF is caused by mutations in the CFTR gene, leading to the absence or dysfunction of the CFTR protein. The most common CFTR mutation — F508del — involves the deletion of three nucleotides (the building blocks of DNA) that impair the ability of the CFTR protein to move salts across the cell membrane. CFTR protein dysfunction in the

WHO Approves Ensifentrine as Name for Potential Inhaled Treatment Now in Clinical Trials

The World Health Organization (WHO) approved ensifentrine as the recommended name to identify the cystic fibrosis (CF) treatment candidate RPL554. Verona Pharma’s ensifentrine is an inhaled inhibitor of the enzymes phosphodiesterase 3 and 4, designed to have bronchodilator and anti-inflammatory properties. The “-fentrine” staple indicates WHO’s recognition that the compound inhibits multiple phosphodiesterases, the company reports. “We are pleased to receive approval from the WHO for the use of ‘ensifentrine’, which to our knowledge, is the only molecule with an INN assignment using the ‘–fentrine’ stem currently in clinical development,” Jan-Anders Karlsson, PhD, Verona’s CEO, said in a pre

Bacterial immunity and infectious disease

Patients with cystic fibrosis are often infected by pseudomonas aeruginosa, a bacterium that infects the lungs and prevents breathing, often causing death. P. aeruginosa itself can also be infected by viruses, which can affect the clinical outcomes of cystic fibrosis patients. Researchers have now used P. aeruginosa as a kind of 'model system' for understanding how bacteria's interactions with viruses may affect human health. Patients with cystic fibrosis are often infected by pseudomonas aeruginosa, a bacterium that infects the lungs and prevents breathing, often causing death. P. aeruginosa itself can also be infected by viruses, which can affect the clinical outcomes of cystic fibrosis pa

Inhalable RNA could breathe new life into lung cancer treatments

Lung epithelial cells that have taken up the mRNA particles (yellow dots), which makes them glow green(Credit: Asha Patel) In the body, messenger RNA (mRNA) molecules are in charge of instructing cells to produce specific proteins, and hijacking this natural system is emerging as a promising new way to treat a wide variety of illnesses. Now, researchers at MIT have developed an inhalable mRNA aerosol that can take the molecules directly to the lungs, as a potential new treatment for cystic fibrosis or lung cancer. Proteins are often called the workhorses of the cell, performing a huge range of functions like transporting molecules or aiding with chemical reactions. Using mRNA to control whic

Online Program Educates Clinicians Treating MS About Common Comorbidities & Current & New Tr

(MENAFN - PRLog) eMultipleSclerosis Review® Volume 2 launches with new design eMultipleSclerosis Review Spread the Word Listed Under Tag: * Cme Industry: * Medical Location: * Manhattan - New York - US Subject: * Features MANHATTAN, N.Y. - Jan. 4, 2019 - PRLog -- DKBmed, LLC, in collaboration with the Johns Hopkins University School of Medicine (JHUSOM), has announced the launch of the second volume of eMultipleSclerosis Review, part of the popular eLiterature Review series that curates and summarizes medical literature and explains how the information applies to clinical practice. eMultipleSclerosis Review Volume 2 has been redesigned with a new, user-friendly interface. This n

Vertex to Request Review of Orkambi, Symkevi Plus Kalydeco for Public Funding in Scotland

Vertex Pharmaceuticals has announced its intention to submit an application to the Scottish Medicines Consortium (SMC) to review the clinical and cost-effectiveness of the company’s cystic fibrosis (CF) medicines, Orkambi (lumacaftor/ivacaftor), as well as Symkevi (tezacaftor/ivacaftor) in combination with Kalydeco (ivacaftor). Following the appraisal of data submitted by Vertex, the SMC will decide whether to accept these CF therapies for use on the National Health Service (NHS) in Scotland, the country’s publicly funded healthcare system. “We are hopeful that, through this process, all eligible patients in Scotland could have access to our medicines soon,” Ludovic Fenaux, senior vice presi

Early detection: Chemists discover new signatures to identify cystic fibrosis in infants sooner with

Scientists at McMaster University have discovered several new biomarkers from a single drop of blood that could allow earlier and more definitive detection of cystic fibrosis (CF), a genetic disease which strikes both children and adults, causing chronic problems with the digestive system and the lungs. The findings, published online in the Journal of Proteome Research, are significant because current newborn screening methods are not accurate enough to identify the disorder in the population, which can manifest itself in many different ways, requiring additional testing and causing further stress for anxious parents. “The earlier CF is detected, the earlier it can be managed and treated, wh

Gene Expression Patterns in Blood May Lead to Patient-tailored CF Treatment, Study Finds

Researchers tested blood samples from cystic fibrosis (CF) patients and identified distinct gene expression patterns that could lead to patient-tailored treatments, a study reports. The study, “Identification of molecular signatures of cystic fibrosis disease status using plasma-based functional genomics,” was published in Physiological Genomics. To better understand CF disease progression and ultimately improve patient care, researchers at Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago gathered blood samples from CF patients and assessed the sequence and levels of a selected set of genes. The genetic data obtained was then associated with

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