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Lying Instead of Sitting During CF Inhalation Therapy Doesn’t Increase Delivery Time, Study Finds

It takes only slightly longer to deliver an inhaled therapy in cystic fibrosis (CF) patients sitting upright than when lying alternately on either side, according to an Australian study. Usually, doctors have patients sit upright when inhaling nebulized medications in order to maximize the lung’s volume. However, experts say distribution patterns of nebulized therapies in the lungs are not uniform in CF patients, with the lungs’ upper lobes receiving lower doses of therapy. The study, “The use of an alternate side lying positioning strategy during inhalation therapy does not prolong nebulization time in adults with Cystic Fibrosis: a randomised crossover trial,” appeared in the journal BMC P

Cystic fibrosis bacterial burden begins during first years of life

Summary: Cystic fibrosis shortens life by making the lungs prone to repeated bacterial infections and inflammation. Researchers have now shown for the first time that the lungs' bacterial population changes in the first few years of life as respiratory infections and inflammation set in. This research offers a way to predict the onset of lung disease in children with CF and suggests a larger role for preventive therapies, such as hypertonic saline. Cystic fibrosis (CF) shortens life by making the lungs prone to repeated bacterial infections and associated inflammation. UNC School of Medicine researchers have now shown for the first time that the lungs' bacterial population changes in the fir

Study offers way to predict onset of lung disease in children with cystic fibrosis

Cystic fibrosis (CF) shortens life by making the lungs prone to repeated bacterial infections and associated inflammation. UNC School of Medicine researchers have now shown for the first time that the lungs' bacterial population changes in the first few years of life as respiratory infections and inflammation set in. The study, published in PLoS Pathogens, offers a way to predict the onset of lung disease in children with CF and suggests a larger role for preventive therapies, such as hypertonic saline. "Lung symptoms in kids with CF are likely due to an increased burden of bacteria," said study senior author Matthew Wolfgang, PhD, associate professor of microbiology and immunology. "This im

Cytomegalovirus-associated pulmonary exacerbation in patients with cystic fibrosis

Abstract CMV is an unusual cause of pulmonary exacerbation in immunocompetent individuals with CF http://ow.ly/Rdds30hlnjV To the Editor: Cystic fibrosis (CF) is a multisystem disease characterised by recurrent respiratory tract infections and progressive respiratory failure. It remains one of the most common life-threatening autosomal recessive conditions affecting Caucasians. Recent studies have highlighted the association between respiratory viral infections and frequency of pulmonary exacerbations, increase antibiotic usage and poorer outcome [1, 2]. Cytomegalovirus (CMV) is a DNA virus and a member of the herpes family of viruses, which includes herpes simplex virus, Epstein–Barr virus

Cystic Fibrosis-Related Diabetes

Cystic fibrosis-related diabetes (CFRD) is a unique type of diabetes that is common in people with CF. With early diagnosis and proper treatment, CFRD can be managed successfully. Causes of CFRD There are two types of diabetes in people without cystic fibrosis. Type 1 diabetes is an autoimmune disease that occurs when the body's immune system attacks and destroys the cells in the pancreas that make insulin (beta cells). Insulin helps the body absorb and use the energy from food. This helps maintain good nutrition and lung function in people with CF. Type 1 diabetes is most commonly diagnosed in childhood. People with type 1 diabetes need to take insulin to stay alive. Type 2 diabetes occurs

Galapagos Provides Update on Cystic Fibrosis Therapies in Development

Galapagos is advancing its development of a range of cystic fibrosis (CF) treatments intended for use in combinations — either with already approved CF therapies or with its own compounds — the Belgian biotech company announced. Phase 2 trials Among the advances is completion of a Phase 2 trial called FLAMINGO (NCT03119649). The study explored a corrector of the CFTR gene (the gene defective in CF), called GLPG2222. CFTR correctors help cells produce a functional CFTR protein even though the gene is mutated. The study included 59 patients with two copies of the F508del mutation in the CFTR gene. All participants had stopped treatment with Orkambi (lumacaftor/ivacaftor) or tezacaftor-ivacafto

2nd Middle East Cystic Fibrosis Conference

MECFC 2018 is taking place in Izmir, Turkey in March. https://lnkd.in/g79mWaP Register today! MECFC 2018 Izmir Türkiye'de yapılıyor. Kaydınızı bugün yaptırın! https://lnkd.in/g79mWaP

Cystic fibrosis drug Orkambi 'approved for use among 6-11 year olds'

The pharmaceutical firm making cystic fibrosis drug Orkambi says it has received EU approval to treat children. Manufacturer Vertex says it will now be suitable for use for those aged 6 to 11. It has already been licensed for patients 12 and over. Earlier this year, the HSE agreed to make Orkambi available to Irish patients after major price negotiations with the pharma industry. According to Vertex, Orkambi is the first medicine to treat the underlying cause of cystic fibrosis (CF) in people with two copies of the F508del mutation. In Europe, there are approximately 3,400 children ages six through 11 with two copies of this mutation. “The innovative long-term agreements we have reached in c

Potential Nitric Oxide Treatment for Resistant Bacterial Infections Gets Patent, May Start Clinical

A possible inhalable treatment for antibiotic-resistant bacterial infections in people with cystic fibrosis due to Pseudomonas aeruginosa now has a U.S. patent and is being readied for a first clinical trial, Novoclem Therapeutics announced. The patent (No. 9,850,322) was issued to the University of North Carolina (UNC) at Chapel Hill where the potential therapy, BIOC51, was discovered, and covers a technology known as water-soluble polyglucosamine compositions that release nitric oxide. Novoclem holds an exclusive license to BIOC51 and will further develop it, including applying to the U.S. Food and Drug Administration (FDA) for the right to test it in people in a controlled study later thi

Introduction of New Therapies Affects Pregnancy Rates in Women With CF, Study Reports

The overall rate at which women with cystic fibrosis are becoming pregnant dropped slightly in recent years — coinciding with the introduction of CFTR modulators and the clinical trials that led to their approval as CF therapies — but appears to be rising again to pre-trial levels, a study reports. The study, “Pregnancy among cystic fibrosis women in the era of CFTR modulators,” appeared in the Journal of Cystic Fibrosis. It calls for more research into the impact of these treatments on maternal and fetal health, and on the effectiveness of contraceptives women of childbearing age, noting a “paucity of data” on these topics. Newer therapies include Kalydeco (ivacaftor) and Orkambi (lumacafto

Cancer gene plays key role in cystic fibrosis lung infections

PTEN is best known as a tumor suppressor, a type of protein that protects cells from growing uncontrollably and becoming cancerous. But according to a new study from Columbia University Medical Center (CUMC), PTEN has a second, previously unknown talent: working with another protein, CFTR, it also keeps lung tissue free and clear of potentially dangerous infections. The findings, published in Immunity, explain why people with cystic fibrosis are particularly prone to respiratory infections—and suggest a new approach to treatment. A quarter-century ago, researchers discovered that cystic fibrosis is caused by mutations in the CFTR gene, which makes an eponymous protein that transports chlorid

Why Aspiration Is a Silent, Hidden Danger for Cystic Fibrosis Patients

Dr. Gwen A. Huitt is an infectious disease doctor at National Jewish Health with a special interest in mycobacteria, bronchiectasis, and cystic fibrosis. Here, she talks to us about the hidden dangers of a major medical issue she feels doesn’t receive the attention it needs in the CF community — aspiration. Q: What is aspiration? What is silent aspiration? A: Aspiration is defined as any liquid, substance, or foreign body that gains access (below the vocal cords) to the airways. Many times when we have an overt aspiration, a cough is triggered. Think, “something went down the wrong pipe.” This may occur when folks drink fluids too quickly, toss their head back to take pills, etc. A small amo

Protein structure could unlock new treatments for cystic fibrosis

Cystic fibrosis is a severe hereditary disease of the lung, for which there is currently no cure. The underlying cause of the disease is a malfunction of the chloride channel CFTR, which prevents the secretion of chloride in certain body cells. This leads to dehydration of the mucus layer in the lung. A promising approach for treating cystic fibrosis is the activation of the calcium-activated chloride channel TMEM16A as an alternative route for chloride efflux. Since TMEM16A is expressed in the same epithelium as CFTR, its activation could restore hydration of the mucus layer. TMEM16A is part of a protein family whose members facilitate the flow of negatively charged chloride ions or lipids

Proteostasis’ CFTR Modulators Show Potential in Improving Effectiveness of Orkambi

Clinical trials on all three Proteostasis Therapeutics’ investigational cystic fibrosis modulators — PTI-428, PTI-801, and PTI-808 — have demonstrated they are safe and have the potential to improve the effectiveness of Orkambi in patients with cystic fibrosis (CF). The positive clinical data supports the initiation of new clinical trials evaluating the drug candidates in doublet and triplet combinations in CF patients. Cystic fibrosis patients have a defective CFTR gene which results in the defective production of CFTR protein — an important channel for the transport of chloride ions in and out of cells, to help control the movement of water in tissues. PTI-428 is a CFTR protein amplifier t

Scientists Explore New Way to Generate Stem Cell-like Cells for CF Therapy

Canadian researchers developed a safer and more cost-effective method for transforming differentiated adult cells into stem-like cells, a process known as induced pluripotent stem cells (iPS). The new method, called interrupted reprogramming, already showed promise as a potential cell therapy replacement in a mouse model with cystic fibrosis. The study “Generation of Induced Progenitor-like Cells from Mature Epithelial Cells Using Interrupted Reprogramming” was published in the journal Stem Cell Reports. “A major block in the critical path of regenerative medicine is the lack of suitable cells to restore function or repair damage,” Thomas Waddell, MD, PhD, co-senior author of the study, said

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