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Tezacaftor, Ivacaftor Combination Generally Safe, Well Tolerated in Children With CF Under Age 12

Data from a phase 3 trial are supporting the use of tezacaftor in combination with ivacaftor for the treatment of cystic fibrosis (CF) in children aged 6 to 11 years, finding that the treatment combination was generally safe and well tolerated. Data from a phase 3 trial support the use of tezacaftor in combination with ivacaftor for the treatment of cystic fibrosis (CF) in children aged 6 to 11 years, finding that the treatment combination was generally safe and well tolerated. Tezacaftor/ivacaftor has previously demonstrated safety and efficacy among patients aged 12 years and older who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation (F/F) or heterozy

Different CFTR Potentiators Bind to Same Flexible Spot on Protein, Study Finds

Approved and investigative cystic fibrosis (CF) treatments known as CFTR potentiators work by binding to the same flexible protein spot — a place that acts like a hinge — and keeping it open, according to a study whose findings may lead to more effective therapies. The research, “Structural identification of a hotspot on CFTR for potentiation,” was published in the journal Science. CF is caused by mutations in the CFTR gene (standing for cystic fibrosis transmembrane conductance regulator), which impairs ion and water transport across cells. This leads to a buildup of thick mucus in organs that include the lungs, and to persistent infections and difficulties breathing. The CF treatment ivaca

Eluforsen Improves CFTR Function in Cellular and Animal Models of CF, Study Says

Eluforsen, an investigational therapy for the treatment of cystic fibrosis (CF), improves the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in cellular and mice models of CF that carry the most common mutation associated with the disorder, a study says. The findings of the study, “Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis,” were published recently in the journal PLOS ONE. CF is a genetic disorder caused by mutations in the CFTR gene, which provides instructions to make the CFTR protein. This protein works as a channel that transports molecules, such as

Kalydeco’s Safety and Benefits Studied in CF Patients with Severe Lung Disease

Treatment with Kalydeco (ivacaftor) improved lung function, exercise capacity, and sweat chloride concentration in cystic fibrosis (CF) patients with severe lung disease, a small retrospective analysis shows. The study, “Effectiveness of ivacaftor in severe cystic fibrosis patients and non‐G551D gating mutations,” was published in the journal Pediatric Pulmonology. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene — the cause of CF — result in the production of a faulty CFTR protein, a “gate” protein that controls the movement of charged salts, such as chloride, in and out of cells. As a result, the mutations cause the “gate” to be stuck closed. Kalydeco is an

Specific Variables Improve Disease Severity Discrimination Among CF Transplant Candidates

The inclusion of Cystic Fibrosis Foundation Patient Registry variables in the lung allocation score (LAS) calculation allowed for enhanced discrimination of disease severity among lung transplant waitlisted candidates with cystic fibrosis (CF), thus providing improved access for those at the highest risk for death. In addition, patients with chronic obstructive pulmonary disease (COPD) also had improved access to transplantation, specifically when forced expiratory volume in 1 second (FEV1) was included as one of the clinical parameters. A multicenter, retrospective, population-based study was conducted among all lung transplant waitlist candidates aged ≥12 years between January 1, 2011, and

Babies Treated with Synagis Show No Lasting Benefits in Lung Health as 7-Year-Olds, Real-world Study

Treating infants with cystic fibrosis (CF) with the antiviral therapy Synagis(palivizumab) does not improve long-term outcomes in lung health, real-world data show. Routine use of Synagis is not recommended by the American Academy of Pediatrics for babies with CF, although its use can be considered in those at risk of chronic lung disease. These findings suggest that for most of these children the therapy does not carry benefits lasting into childhood. The study “Palivizumab and Long-term Outcomes in Cystic Fibrosis” was published in the journal Pediatrics. Synagis, marketed by AstraZeneca, is an engineered antibody designed to block the proliferation of cells infected by the respiratory syn

Organoids grown in microfluidic device may help cf patients with diabetes

Scientists created human pancreas on a chip that allowed them to identify the possible cause of a frequent and deadly complication of cystic fibrosis (CF) called CF-Related Diabetes, or CFRD. It may be feasible to also use the small two-chambered device, which features bioengineered human pancreatic organoids to study the causes of non-CF-related conditions such as type 1 and 2 diabetes, according to researchers at Cincinnati Children's Hospital Medical Center, who report findings in Nature Communications. First, however, the scientists want to see if their device can help people with CF—a genetic lung disease caused by a mutation in the CFTR gene. The mutation leads to a water and salt imba

Modified and More Rigorous Cleansing Procedure May Better Prepare CF Patients for Colonoscopy, Study

People with cystic fibrosis (CF) may require a modified and more rigorous cleansing procedure to prepare their bowels for a colonoscopy screening procedure that allows good visualization, a study reports. Findings of the study, “A non-randomised single centre cohort study, comparing standard and modified bowel preparations, in adults with cystic fibrosis requiring colonoscopy,” were published in the journal BMC Gastroenterology. CF is a genetic disorder caused by mutations in the CFTR gene, which provides instructions to make the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Although CF is mostly associated with respiratory symptoms, gastrointestinal (GI) complications

Combining Antibiotics, Researchers Deliver One-Two Punch Against P. aeruginosa

By combining two well-established antibiotics for the first time, a scientific team led by Case Western Reserve University School of Medicine and Louis Stokes Cleveland VA Medical Center has delivered a "double whammy" against the pervasive Pseudomonas aeruginosa, a potentially deadly form of bacteria that is a major source of hospital-based infections. In a recent Journal of Infectious Diseases study, investigators showed using two antibiotic drugs to fight P. aeruginosa in mouse models was significantly more effective than either antibiotic alone. The antibiotics were ceftazidime-avibactam, a combination drug used to treat a wide variety of serious bacterial infections, and fosfomycin, use

AiCuris and Lysando Join Forces in the Fight Against Antimicrobial Resistance

Cooperation focused on the joint development and optimization of human anti-infective drug candidates against gram-negative and gram-positive bacteria in relevant indications using Lysando's Artilysin(R)s technology platform Artilysin(R)s represent a completely new class of molecules with a novel mode of action with the potential to replace conventional antibiotics; platform already validated in veterinary medicine and medical devices Unparalleled expertise in anti-infective drug development best positions AiCuris to exploit the potential of Lysando's Artilysin(R) technology platform in the fight against antimicrobial resistance WUPPERTAL, GERMANY and BALZERS, LIECHTENSTEIN / ACCESSWIRE / Ju

Hinge-like protein may open new doors in cystic fibrosis treatment

In recent decades, treatment options for people with cystic fibrosis have improved dramatically. The newest drugs, known as potentiators, target a protein called cystic fibrosis transmembrane conductance regulator, which is mutated in people with the disease. Yet, while these medications can help some people with CF, they are far from perfect. Moreover, researchers haven't been able to figure out how the drugs actually work—until now. A new study by Rockefeller scientists characterizes, for the first time, the interaction between potentiators and the protein they target at atomic resolution. The research, described in a recent report in Science, shows that two distinct compounds act on the s

Ultrasound Can Help Detect Liver Disease in Kids with CF, Study Says

Ultrasound imaging can be a reliable diagnostic tool for early detection of liver disease in children with cystic fibrosis (CF), a study finds. Using this strategy can help clinicians identify CF patients most at risk for liver damage caused by cirrhosis (tissue scarring), researchers suggest. These findings were reported in the study, “Liver ultrasound patterns in children with cystic fibrosis correlate with non-invasive tests of liver disease,” published in the Journal of Pediatric Gastroenterology and Nutrition. Cirrhosis, which refers to late-stage of fibrosis of the liver, occurs in approximately 5–7% of people with CF and is the third-leading cause of death in these patients. There is

Better Access to Relizorb for Patients on Feeding Tubes Advocated by Doctors and Dietitians

More than 60 cystic fibrosis experts strongly support Relizorb being made available to CF patients on tube feeding, pushing in a commentary for the insurance coverage that’s generally denied this digestive enzyme cartridge, and inviting “the CF community” to become advocates of better patient access to the device. Their opinion piece included a comprehensive analysis of Relizorb’s benefits, based on studies, reviews, and these experts own real-world evidence, collected from about 450 of their patients using the Alcresta Therapeutics‘ device. “[T]he signatories of this communication … view this as a valuable therapeutic option for their CF patients who require tube feedings,” the group wrote.

Hypertonic saline may be appropriate intervention for preschoolers with cystic fibrosis

New data from the SHIP study show that inhaled hypertonic saline, as compared with isotonic saline, improved lung clearance index in children aged 3 to 6 years with cystic fibrosis. Specifically, at 48 weeks, children randomly assigned hypertonic saline experienced a significant decrease in lung clearance index (LCI2.5) when compared with children assigned isotonic saline, with a mean treatment effect of –0.63 LCI2.5 units (95% CI, –1.1 to –0.15). Results were similar in sensitivity analyses, the researchers reported. Also at 48 weeks, Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain scores declined in both the hypertonic saline and isotonic saline groups, with no significant

BillionToOne Announces Launch of UNITY Prenatal Screen

The diagnostics company BillionToOne has launched its first product for commercial and clinical use — a blood test designed to help diagnose hereditary diseases in fetuses. The new product, called UNITY, tests cell-free fetal DNA to aid in the diagnosis of cystic fibrosis (CF), spinal muscular atrophy, sickle cell disease, and thalassemias, all of which are medical conditions recommended for testing by the American College of Obstetricians and Gynecologists (ACOG). Generally, screening for these conditions has been done by testing the biological parents to see whether they are carriers of mutations causing the diseases. However, this method is imperfect for several reasons. It doesn’t actual

FDA Grants Orphan Drug Status to AR-501, Potential CF Treatment for Chronic Lung Infections

AR-501, an investigational inhaled formulation of gallium citrate to treat chronic lung infections in patients with cystic fibrosis (CF), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The treatment, developed by Aridis Pharmaceuticals, is designed to be given once weekly and is self-administered within minutes, allowing a direct delivery to the lungs. The safety and preliminary efficacy of AR-501 is currently being evaluated in an ongoing Phase 1/2 trial in healthy volunteers and CF patients with chronic lung infections due to Pseudomonas aeruginosa. Orphan Drug Designation is granted to potential treatments that demonstrate promise for the diagnosis or

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