News & Articles

Second-hand Smoke Affects Metabolism in Infants and Children with CF, Study Finds

Exposure to second-hand smoke in infants and children with cystic fibrosis (CF) is linked to changes in levels of metabolites and activation of signaling pathways that enhance inflammation, a study has found. The findings suggest exposure to second-hand smoke is a potentially important environmental factor in CF. The study “Metabolomics profiling of tobacco exposure in children with cystic fibrosis” was published in the Journal of Cystic Fibrosis. Environmental factors are known to influence the course of inflammation and infections in CF patients. In previous studies, researchers from Nationwide Children’s Hospital in Columbus, Ohio, found that exposure to second-hand smoke was associated w

Cystic Fibrosis and Herpes Are Not the Same Thing

I don’t have herpes. I need to clarify this. I don’t have herpes, or Hep C, or cancer, even though many seem to think I do. Although I empathize with the Herps and Hep sufferers of the world, I am going to lose my mind if another person comments on health-related posts to suggest magician-doctor-cults or modified-herb-mojos to cure me. “Have you tried celery juice?” This was the direct message I received today in response to an Instagram story reporting my latest pain attack by my pancreas and liver. I shared this information because I figured the complete lack of dancing footage would make the sudden lifestyle shift obvious sooner or later. I didn’t reply because I don’t want to be mean. Ma

4DMT Receives Up to $14M from CFF for Development of Customized Gene Carrier

After being given more than $3 million from the Cystic Fibrosis Foundation (CFF) in 2017 to expand the development of viral vectors for gene delivery in the lungs, 4D Molecular Therapeutics (4DMT) now will receive up to $14 million from CFF to support the development of a customized gene carrier for the treatment of cystic fibrosis (CF). The new gene carrier, called 4D-710, is a harmless adeno-associated viral vector (AAV) designed to deliver a functional copy of the CFTR gene — the faulty gene in people with CF — directly to patient lung cells. “Today’s announcement reaffirms our commitment to invest aggressively to draw the best scientific minds and technology into CF and deliver the next

Bacterial 'Death Stars' could be tricked into destroying themselves

Researchers have discovered a network of channels inside bacterial communities which could be used to kill bacteria more quickly by 'tricking' them into transporting drugs. The communities—called biofilms—are involved in up to 80% of persistent human infections and cannot be killed easily by antibiotics. Researchers in the Department of Physics and the Strathclyde Institute for Pharmacy and Biomedical Sciences at the University of Strathclyde have found that when the bacterium E. coli is living as a biofilm, it forms channels to help transport nutrients around its community. They discovered these channels to transport nutrients into the biofilm could also be exploited to design new methods f

Kalydeco Approved in Europe to Treat Children with R117H Mutation

The European Commission has approved Vertex’s Kalydeco (ivacaftor) for the treatment of children and adolescents with cystic fibrosis (CF) who carry the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is defective in CF. Kalydeco previously was approved in Europe for children (6 months and older) and adolescents with at least one of nine gate-closing mutations: G551D, G1244E, G1349D, S549R, G178R, S1251N, S1255P, S549N, and G551S. The therapy also is approved for CF patients with the R117H mutation who are 18 and older. The new label extension allows patients 6 months and older with the R117H mutation and who weigh at least five kilograms (about 1

Suppressing Function of Chloride Channel TMEM16A Eyed as Potential Therapeutic Approach

Researchers have reported the case of two siblings with impaired CFTR function due to a deficiency in another chloride channel, called TMEM16A, that do not present symptoms associated with cystic fibrosis (CF). While there is evidence supporting either the activation or the suppression of TMEM16A activity as a potential treatment of CF, these findings support the latter. Clinical trials evaluating the therapeutic benefit of blocking TMEM16A function in CF patients are on the horizon, researchers believe. The study, “TMEM16A deficiency: a potentially fatal neonatal disease resulting from impaired chloride currents,” was published in the Journal of Medical Genetics. CF is caused by a deficienc

Microbe DNA Discovery May Lead to Noninvasive Test for CF Infections

People with cystic fibrosis (CF) have higher levels of DNA from common microbes that cause CF-related infections circulating in their blood compared to healthy controls, a study discovered. These findings support the development of a simple blood test as a noninvasive diagnostic tool to identify chronic lung infections in CF patients. The study, “Cell free DNA from respiratory pathogens is detectable in the blood plasma of Cystic Fibrosis patients,” was published in the journal Nature Scientific Reports. CF is marked by chronic airway infection. Accurately diagnosing and monitoring the start of infection can be challenging, especially in children who often are unable to provide regular sputu

Real-world Use of Kalydeco for CF Highly Effective, Large-scale Analysis Shows

Kalydeco treatment led to fewer deaths, transplants, hospitalizations, and exacerbations in people with cystic fibrosis (CF) compared to patients who did not receive the therapy, according to a five-year, real-world analysis of patient registries in the U.S. and U.K. The results of the analysis were published in the journal Pulmonary Therapy, under the title, “Real-World Outcomes Among Patients with Cystic Fibrosis Treated with Ivacaftor: 2012–2016 Experience.” CF is caused by mutations in the CFTR gene that result in a faulty CFTR protein that controls the flow of charged salts (ions), such as chloride, in and out of cells. While some mutations lead to a completely inactive CFTR protein, mu

Pharmac funds new cystic fibrosis medicine

New treatment for cystic fibrosis is on the horizon, one that will help young children suffering from the disorder digest their food more easily. Government drug-buying agency Pharmac announced on Saturday the drug Creon Micro will be available from Monday. Pharmac says it could benefit about 60 Kiwis suffering from cystic fibrosis - a rare disorder that damages the digestive system and lungs. Cystic Fibrosis New Zealand chief executive Jane Bollard said in a statement the medication will mean a happier baby and family. "The first years following diagnosis of cystic fibrosis are a stressful time for new parents and anything that can ease the load on families and whānau is most welcome." It c

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred in Stockholm, but instead took place online via Zoom. “We’re interested in this topic because our community feels poorly served by newborn screening,” said Nick Meade, policy director at Genetic Alliance UK, a national charity representing more than 230 British patient advocacy organizations. Meade said a newborn screening “patient charter” — launched in July 2019 — has already been endorsed by so

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