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Vertex and Republic of Ireland Reach Agreement on Reimbursement for Triple Combination Therapy

Patients with cystic fibrosis (CF) in the Republic of Ireland will be among the first in Europe to benefit from Vertex Pharmaceuticals’ triple combination regimen composed of elexacaftor (VX-445), tezacaftor, and Kalydeco (ivacaftor), upon its approval by the European Medicines Agency. Following Vertex’s negotiations with the Republic of Ireland’s Health Service Executive, the company’s triple combination regimen has been added to the existing long-term CF reimbursement agreement. The triple combination regimen is currently being reviewed by the European Medicines Agency (EMA), and is awaiting approval for all indicated CF patients who are at least 12 years old. Of note, this triple regimen

Care of Children with Bronchiectasis Lags Those with Cystic Fibrosis, Australian Study Finds

Children with bronchiectasis have poorer lung function than those with (CF), likely because of the u8differing levels of care given to children with these conditions, an Australian study suggests. The study, “Children with bronchiectasis have poorer lung function than those with cystic fibrosis and do not receive the same standard of care,” was published in the journal Pediatric Pulmonology. Bronchiectasis and CF both can seriously affect the lungs’ ability to work; in fact, people with CF can develop bronchiectasis as a secondary complication. Detailed guidelines for how best to provide medical care for children with CF are available. Typically, these involve a multidisciplinary team, and h

The immune system listens in on bacteria signaling to each other

The immune response can be modulated by bacterial signaling molecules. One bacterium does not an infection cause; the microbes work as a team. Some species send and receive molecular signals so they can keep tabs on how many fellows surround them, a process known as quorum sensing. Virulence kicks into high gear when this signaling indicates that the bacterial population reaches a certain density threshold—the requisite quorum. One species that relies on quorum sensing is P. aeruginosa, a nasty pathogen that infects and causes disease in plants, animals and, when an opportunity presents itself, in humans. It is a leading cause of morbidity and mortality in burn victims, people with cystic fi

IGF2R Blood Levels May Help Predict Antibiotic Response in CF Pulmonary Exacerbations, Study Says

Measuring the levels of insulin-like growth factor 2 receptor (IGF2R) during the first days of treatment with intravenous antibiotics could help predict treatment response in cystic fibrosis patients experiencing pulmonary exacerbations, a study has found. The study, “Identification of novel blood biomarkers of treatment response in cystic fibrosis pulmonary exacerbations by label-free quantitative proteomics,” was published in the journal Nature Scientific Reports. Pulmonary exacerbations are frequent among people with cystic fibrosis (CF). They are commonly caused by viral and bacterial infections, but can also result from exposure to non-infectious factors, such as polluted air. The most

Proteostasis to Test Personalized Treatment with Novel CFTR Modulators in Europe

Three investigative therapies by Proteostasis Therapeutics — PTI-801, PTI-808, and PTI-428 — showed potential to treat cystic fibrosis (CF) patients ineligible for approved CFTR modulators after patient-specific lab models produced promising results that support a future clinical trial. Funded by the EU’s initiative HIT-CF (Human Individualized Therapy of CF), this early study was conducted on patient organoids, which are miniaturized organ models derived from patient cells. Based on the positive results, a clinical trial in adult CF patients is expected to start in 2020. If successful, the trial results may serve as the basis for a marketing authorization application in Europe in 2021. With

Approval of drug therapy a 'new dawn' for cystic fibrosis care in Ireland

A drug which has the potential to treat the underlying cause of cystic fibrosis for 80% of patients in Ireland has been approved by the HSE. The Trikafta drug therapy will be made available to eligible patients once it has been approved by the European Medicines Agency. The triple combination treatment is not currently approved for use in Ireland. It is the first approved treatment that is effective for people with cystic fibrosis who are aged 12 and over and who have at least one F508del mutation. 90% of patients worldwide and 80%, or 1,000 people, in Ireland are affected by this mutation. Philip Watt, CEO of Cystic Fibrosis Ireland said the approval was "a most wonderful Christmas present

Medical News Today: Celiac disease may be treated with cystic fibrosis drugs

New research investigates the molecular similarities between celiac disease and cystic fibrosis. The results suggest that a compound developed for cystic fibrosis may also treat celiac disease. New research may bring us closer to a treatment for celiac disease. is an autoimmune disorder that affects people in the United States. The condition is triggered by the consumption of gluten — a protein that can be found in wheat, barley, and rye and in foods such as bread, pasta, and baked goods. In a person with celiac disease, consuming gluten causes the immune system to attack the mucus that lines the inside of the small intestine. This can trigger a range of digestive symptoms, such as bloating,

New technology for protein bioconjugation and structural proteomics

Researchers at the Czech Academy of Sciences and the Brno-based start-up CF Plus Chemicals, a spin-off of ETH Zurich, has reported a new technology called CF LINK for site-selective bioconjugation of proteins and their structural characterization. The technology can selectively prepare protein conjugates via their tryptophan residues and perform post-translational modification of aromatic amino acids. Furthermore, it can also be used as a tool for the mapping of protein surfaces and studies of protein-protein interactions. The company CF Plus Chemicals, an ETH Zurich spin-off founded in 2014, is based on almost 10 years of cooperation of the group of Dr. Petr Beier at the Institute of Organi

What are the benefits of NAC?

N-acetylcysteine (NAC) is a supplement that has been available for several decades. People take it for various reasons, including to help treat medical issues ranging from psychological disorders to chronic lung conditions and to improve athletic performance. Doctors believe that NAC may stimulate the synthesis of glutathione — a compound that helps fight free radicals, unstable atoms that can cause inflammation and damage. Keep reading to learn how NAC may benefit the body. We also describe risks, including side effects, associated with the supplement. Benefits Researchers have investigated the potential for NAC to help treat a wide variety of health issues. Some research indicates that NAC

Israeli, US Researchers Develop New Device to Eliminate Mucus Secretion from Obstructed Airways

Researchers in Israel and the U.S. have developed a new technology that can be used to unclog and eliminate mucus secretions from the airways of patients with various respiratory diseases, including cystic fibrosis (CF). The announcement was made by BGN Technologies, the technology company of Ben-Gurion University (BGU), which markets innovations developed in its lab. The new tech was developed in a collaboration between BGU’s David Katoshevski, PhD, from the unit of environmental engineering, and Yuval Cavari, MD, of Soroka Medical Center’s pediatric intensive care unit, both in Israel, working together with University of Cincinnati’s Ephraim Gutmark, PhD, from the college of engineering an

New Tool for Rapidly Analyzing CRISPR Edits Reveals Frequent Production of Unintended Edits

New method unmasks all DNA changes caused by CRISPR in two days vs two months WILMINGTON, DE — Amidst rising hopes for using CRISPR gene editing tools to repair deadly mutations linked to conditions like cystic fibrosis and sickle cell disease, a new study in the Nature journal Communications Biology describes a new innovation that could accelerate this work by rapidly revealing unintended and potentially harmful changes introduced by a gene editing process. "We've developed a new process for rapidly screening all of the edits made by CRISPR, and it shows there may be many more unintended changes to DNA around the site of a CRISPR repair than previously thought," said Eric Kmiec, PhD, direct

IGF2R Blood Levels May Help Predict Antibiotic Response in CF Pulmonary Exacerbations, Study Says

Measuring the levels of insulin-like growth factor 2 receptor (IGF2R) during the first days of treatment with intravenous antibiotics could help predict treatment response in cystic fibrosis patients experiencing pulmonary exacerbations, a study has found. The study, “Identification of novel blood biomarkers of treatment response in cystic fibrosis pulmonary exacerbations by label-free quantitative proteomics,” was published in the journal Nature Scientific Reports. Pulmonary exacerbations are frequent among people with cystic fibrosis (CF). They are commonly caused by viral and bacterial infections, but can also result from exposure to non-infectious factors, such as polluted air. The most

5 New Drug Approvals the FDA Gave Top Priority

The U.S. Food and Drug Administration's made it clear that granting a priority review to an application isn't exactly the same thing as making an application's review a top priority. Over the past five weeks, the agency stamped approvals on five new drugs months ahead of an already shortened schedule. You'll be relieved to know the FDA doesn't allow biopharmaceutical companies to cut in line without good reasons. If you'd like to know more about what works, have a look at some things they have in common. First-timers Developing first-in-class new drug candidates for life-threatening illnesses that lack treatment options is the best way to get the FDA's attention. Four of these new drugs are

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