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Case Report Cites IDPN as Nutritional Tool in CF Patients with Kidney Disease

Delivering nutrients through dialysis could help people with co-occurring cystic fibrosis (CF) and kidney disease meet their nutritional requirements, a recent case report illustrates. The report, “Intradialytic parenteral nutrition improves nutritional status in a complex cystic fibrosis patient with redo double lung transplant and end-stage renal disease,” was published in the journal BMJ Case Reports. Dialysis is a medical procedure that replicates some of the functions of the kidneys (e.g., filtering the blood), and is used to help manage kidney failure. Intradialytic parenteral nutrition (IDPN) refers to nutrients being added to the blood during dialysis. IDPN can be helpful when a pers

Protein Called TAS2R38 Linked to Respiratory Tract Complications in CF

Problems with a protein called TAS2R38 are linked to a greater risk of severe sinonasal disease and Pseudomonas aeruginosa lung infections early in life in people with cystic fibrosis (CF), a study reported. These findings may help to identify high-risk patients, those with a dysfunctional form of TAS2R38, and improve approaches taken to prevent these respiratory tract complications. The study, “TAS2R38 is a novel modifier gene in patients with cystic fibrosis,” was published in the journal Nature Scientific Reports. CF is caused by mutations in the CFTR gene, which result in low to no production of CFTR, a protein that controls secretion of body fluids such as mucus. The deficiency leads to

Staphylococcus Aureus in CF Airways Change With Age, Antibiotic Use, Study Says

Different types of Staphylococcus aureus can be found persistently in the airways of cystic fibrosis (CF) patients, but this bacteria population is influenced by age and prior antibiotic treatment, a new study says. The study, “Antibiotic Treatment and Age Are Associated With Staphylococcus aureus Carriage Profiles During Persistence in the Airways of Cystic Fibrosis Patients,” was published in the journal Frontiers in Microbiology. People with CF often experience chronic bacterial infections of the airways, which can lead to decreased lung function. S. aureus is one of the earliest bacteria present in the airways of people with CF, as early as infancy. S. aureus is known to persist in the a

Planned CHOICES Trial in Rare CF Mutations Given Regulatory Support

Proteostasis Therapeutics reported receiving scientific advice for its CHOICES clinical trial program, which is to test its personalized triple combination for cystic fibrosis (CF) patients without available treatment options, from the Dutch Medicines Evaluation Board. Regulatory officials outlined necessary steps in this planned, pivotal trial that would support approval of the company’s CFTR modulators — dirocaftor, posenacaftor, and nesolicaftor — if results are favorable. They expressed support for the company’s goal of helping patients which the rarest forms of CF, Proteostasis reported in a press release. But they also voiced interest in expanding this personalized treatment approach t

Simulations show how to make gene therapy more effective

Visualisation of a lipoplex entrapped inside an endosome. The DNA (yellow/pink rods) can only escape upon fusion of the lipoplex (red) with the endosomal membrane (blue). Credit: Marrink Lab, UG Diseases with a genetic cause could, in theory, be treated by supplying a correct version of the faulty gene. However, in practice, delivering new genetic material to human cells is difficult. A promising method for the delivery of such genes involves the use of DNA/lipid complexes (lipoplexes). Scientists at the University of Groningen have now used advanced simulations to investigate how these lipoplexes deliver DNA fragments into cells. The results, which were published in the journal eLife on 16

Breaking the Diagnosis of Cystic Fibrosis to Parents: A process not a one-off event

2019 Royal Society of Medicine Cystic Fibrosis Symposium Breaking the Diagnosis of Cystic Fibrosis to Parents: A process not a one-off event Abstract Breaking the news to parents that their child has cystic fibrosis [CF] is most frequently given in the first few weeks of the baby’s life as a result of newborn screening. This is optimal to reduce morbidity but can have a significant impact on the parents’ mental wellbeing and the parent-child relationship. Parent feedback indicates that assimilating the diagnosis is not a one-off event but a process that takes time. CF professionals therefore need to be aware not only of how they communicate the diagnosis initially but also the ways in which

Coronavirus update: More considered vulnerable - full list of who should stay at home

CORONAVIRUS can be dangerous for the most vulnerable members of society. As time goes on, more health conditions - that increase a person's risk of serious consequences from COVID-19 - are being identified. Coronavirus lockdown may be extended as society tries to grapple with the pandemic. New health conditions have been added to the list that requires people to stay at home for three months. What are they? On Tuesday April 7, at the Downing Street briefing, Chief Medical Officer Professor Chris Whitty commented on shielding measures. He said: "There are additional people who have been identified either by specialist medical groups or by GPs. "[They] know that someone has got a group of cond

Arrowhead Pharmaceuticals files for regulatory clearance to begin phase 1/2a study of ARO-ENaC for t

rrowhead Pharmaceuticals has filed an application for clearance to begin a Phase 1/2a clinical trial of ARO-ENaC, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with cystic fibrosis (CF), which is a rare disease caused by genetic mutations that lead to progressive deterioration in lung function due to poor clearance of mucus and associated recurrent infections. Arrowhead Pharmaceuticals files for regulatory clearance to begin phase 1/2a study of ARO-ENaC for treatment of cystic fibrosis. (Credit: PublicDomainPictures from Pixabay) ARO-ENaC utilizes Arrowhead’s proprietary Targeted RNAi Molecule (TRiM) platform and is the company’

McGill spinoff Laurent Pharmaceuticals to begin clinical trial on COVID-19 treatment

Company to run a Phase 2 clinical study with LAU-7b, a pro-resolving drug with potential antiviral properties against coronavirus Junji Nishihata, Senior Communications Adviser, Office of Research and Innovation Laurent Pharmaceuticals Inc., a clinical-stage biopharmaceutical company that spun off from McGill in 2012, announced on April 9, that it is planning to test its lead drug LAU-7b in patients with COVID-19 disease. LAU-7b was recently identified as a potential anti-viral therapeutic option for COVID-19 during a drug-library screening effort conducted by scientists from the Institut Pasteur Korea. The company is seeking approvals for clinical trials in Canada and the US. Currently bein

Verona Pharma Reports Positive Efficacy and Safety Data with Single Dose pMDI Formulation of Ensifen

Statistically significant and clinically meaningful improvements in lung function Ensifentrine has now demonstrated positive efficacy and safety in COPD patients via three widely used inhalation modes: nebulizer, DPI and pMDI Initiation of multiple dose part of pMDI trial postponed due to the COVID-19 situation LONDON, March 31, 2020 (GLOBE NEWSWIRE) -- Verona Pharma plc (AIM: VRP) (Nasdaq: VRNA) (“Verona Pharma”), a clinical-stage biopharmaceutical company focused on respiratory diseases, announces positive efficacy and safety data with a single dose of pressurized metered-dose inhaler (“pMDI”) formulation of ensifentrine in a Phase 2 clinical trial in patients with moderate to severe chron

UHG Adult Cystic Fibrosis Team to use online videos for outpatient consultations

Galway Bay fm newsroom: The Adult Cystic Fibrosis Team at UHG will use online video conferencing instead of face-to-face outpatient appointments during the COVID-19 outbreak. The team began work on its telemedicine project at the start of this year, which involves delivering a physiotherapy service to patients online. The video link technology allows patients to speak with physicians, the clinical nurse specialist, the dietitian and a physiologist as required. The video consultation covers everything patients would normally get from a face-to-face meeting, apart from respiratory testing and measuring of vital signs. Plans are being formulated to conduct these types of tests remotely in the f

CF Patients’ Risk For Ototoxicity Underreported

Las Vegas—The complex needs of patients with cystic fibrosis (CF) who are managed by specialty pharmacies just got a bit more complicated by a new study showing that these patients have a much higher risk for developing aminoglycoside-induced ototoxicity than previously thought. The researchers are urging centers to monitor patients with CF more closely for aminoglycoside-induced ototoxicity after finding high rates of the complication at their institution. After implementing a pharmacist-recommended monitoring protocol, physicians increased their use of audiograms in at-risk patients from roughly 25% to nearly 100% and found more than 55% were experiencing aminoglycoside-induced ototoxicity

Study Finds Funding Disparities Between Sickle Cell Disease, Cystic Fibrosis

Disparities in funding exist between sickle cell disease (SCD) and cystic fibrosis (CF), according to a new study published in JAMA Network Open. For SCD, the disparities may be associated with decreased research productivity and novel drug development. In this cross-sectional study, researchers compiled data on funding and outputs for CF and SCD. Specifically, they assessed whether expenditures by the National Institutes of Health (NIH) and each diseases’ national foundations are associated with the number of publications indexed in PubMed, US FDA drug approvals, and active clinical trials. NIH funding is allocated in accordance with disease burden. Because heart disease and cancer are

Student Project Aims to Make CF Physiotherapy a Game

A video game aimed at encouraging young cystic fibrosis (CF) patients to do their breathing exercises is being tested at Great Ormond Street Hospital in London and other sites across the UK Konglomerate Games, a company comprised of fourth-year students from Abertay University in Scotland, developed the project using £10,000 ($11,600) in funding from the Unloc Enterprise Challenge, a Europe-wide online entrepreneurship competition for individuals ages 16 to 25. The game is crafted around the Project Fizzyo device, a bluetooth sensor that connects to physiotherapy tools patients use to help clear airways. Removing mucus is very important to prevent airway infections in people with CF, and to

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