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Kalydeco’s Efficacy Tied to Phosphorylation Levels of CFTR Protein, Study Suggests

CFTR channel activity is controlled by a molecular process known as phosphorylation. Now, a study suggests that the levels of phosphorylation affect how well Kalydeco works in people with certain mutations in the CFTR gene. The study “VX-770-mediated potentiation of numerous human CFTR disease mutants is influenced by phosphorylation level” was published in the journal Nature Scientific Reports. More than 2,000 mutations have been identified in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, with evidence linking 300 of these mutations with the development of cystic fibrosis (CF). The gene has instructions for the production of the CFTR protein, which serves as a gate at

New protein could offer a fresh target for cancer treatment

An international study in the Proceedings of the National Academy of Sciences shows a fascinating new drug target – a cell protein dubbed mitoNEET that determines how materials are transported from the mitochondria to the rest of the cell. The mitochondria are tiny bean-shaped parts of almost every cell that are responsible for supplying power for all cellular functions – the powerhouses of the cell. They draw out the energy in food molecules and convert it to usable chemical energy. The mitochondria are bounded by inner and outer membrane layers that isolate them from the rest of the cell. These membranes are perforated by multiple pores, formed by specially designed proteins called porins

Eloxx Pharmaceuticals Announces Cystic Fibrosis Foundation (CF Foundation) To Provide Funding and Su

CF Foundation and Eloxx to form joint program advisory group focused on the development of ELX-02 for cystic fibrosis Eloxx plans to conduct the U.S. portion of the Phase 2 clinical trial at Cystic Fibrosis Therapeutics Development Network (TDN) member sites On track to report top line Phase 2 data for ELX-02 in cystic fibrosis and cystinosis in 2019 Eloxx to host one-on-one meetings with institutional investors at the Oppenheimer Fall Summit focused on Specialty Pharma & Rare Diseases (September 24, 2019) and to present and host one-on-one meetings at the 2019 Cantor Global Healthcare Conference (October 3, 2019) WALTHAM, Mass., Sept. 23, 2019 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc

Safety, Early Effectiveness of POL6014 Confirmed in Phase 1 Trial

Single dosing of orally inhaled POL6014, an experimental therapy to treat chronic lung inflammation, can effectively block the activity of a pro-inflammatory enzyme in the lungs of people with cystic fibrosis (CF), results from a Phase 1 clinical trial show. The data were published in the Journal of Cystic Fibrosis in the study, “Single dose escalation studies with inhaled POL6014, a potent novel selective reversible inhibitor of human neutrophil elastase, in healthy volunteers and subjects with cystic fibrosis.” Neutrophils, a type of immune cells, form part of the primary line of defense of the immune system in case of infection. These cells release an enzyme, called neutrophil elastase (N

Amikacin Liposome Therapy Has Similar Efficacy as Tobramycin for P. aeruginosa, Study Shows

Amikacin liposome inhalation suspension (ALIS) showed the same efficacy and similar safety profile as tobramycin inhalation solution (TIS) for the treatment of cystic fibrosis (CF) patients with Pseudomonas aeruginosa lung infection, a comparative clinical study showed. The study, “Amikacin liposome inhalation suspension for chronic Pseudomonas aeruginosa infection in cystic fibrosis,” was published in the Journal of Cystic Fibrosis. CF patients often have chronic P. aeruginosa lung infections. These bacteria infect approximately 30% of CF patients ages 6–10, and 60% of CF patients ages 18–24. Compared to other CF pathogens, P. aeruginosa is difficult to eradicate and can lead to clinical de

National Study to Use Telehealth for Talk Therapy for CF Patients

Led by researchers at Drexel University, a national study will evaluate whether talk therapy delivered via telehealth can help people living with cystic fibrosis who struggle with anxiety and depression. September 06, 2019 - Researchers at several health systems are studying whether talk therapy delivered by telemedicine can alleviate anxiety and depression in people living with cystic fibrosis and thus improve care management and clinical outcomes. The project is being spearheaded by C. Virginia O’Hayer, PhD, a clinical associate professor of psychiatry at Philadelphia-based Drexel University’s College of Medicine, and follows a three-year pilot conducted by O’Hayer in which telehealth redu

Cystic fibrosis carriers: What to know

A person who is a cystic fibrosis carrier has one copy of the gene for the condition. People need to have two copies of the gene to have cystic fibrosis. If a cystic fibrosis (CF) carrier has a child with another CF carrier, there is a chance that the child may also be a carrier or that they might have the disease. Research suggests that as many as 10 million people in the United States are CF carriers. Testing can help an individual discover whether they are among these individuals. Read on to learn more about cystic fibrosis carriers. Signs you may be a carrier CF carriers do not experience any symptoms. As a result, most people who are carriers remain unaware that they have the CF gene. A

Human pancreas on a chip can reveal more about disease

A researcher holds a dual-chamber chip that scientists used to bioengineer human pancreatic tissues to study the cause of Cystic Fibrosis-Related Diabetes, a frequent and deadly complication for people who battle the genetic lung disease cystic fibrosis. The scientists report data from their study in Nature Communications. It might be sensible to likewise utilize the little two-chambered gadget, which highlights bioengineered human pancreatic organoids to overview the reasons for non-CF-related conditions, for example, type 1 and 2 diabetes, as indicated by agents at Cincinnati Children’s Hospital Medical Center, who report discoveries in Nature Communications. In the first place, be that as

Aridis Pharmaceuticals Reports Phase 2 Clinical Trial Results of AR-105 for the Treatment of Ventila

SAN JOSE, Calif., Sept. 3, 2019 /PRNewswire/ -- Aridis Pharmaceuticals, Inc. (Nasdaq: ARDS), a biopharmaceutical company focused on the discovery and development of novel anti-infective therapies to treat life-threatening bacterial infections, today announced results from the Company's first-in-patient Phase 2 clinical trial evaluating AR-105, a fully human IgG1 monoclonal antibody for the treatment of ventilator-associated pneumonia (VAP) caused by gram-negative Pseudomonas aeruginosa (P. aeruginosa). The recently completed study did not meet its primary endpoint of demonstrating superiority in Clinical Cure rates on Day 21 compared to placebo. Furthermore, there was a statistically signifi

FDA to Review Elexacaftor, Tezacaftor, Ivacaftor Combo for Cystic Fibrosis

The Food and Drug Administration (FDA) has accepted for priority review the New Drug Application (NDA) for elexacaftor (VX-445), tezacaftor and ivacaftor triple combination regimen for patients with cystic fibrosis. Elexacaftor is an investigational therapy, while the combination of tezacaftor and ivacaftor and ivacaftor alone are both already approved treatments for cystic fibrosis. The NDA submission includes data from two phase 3 trials assessing the triple combination therapy in patients with one F508del mutation and one minimal function mutation, as well as those with two F508del mutations. Results from both studies showed that treatment with elexacaftor, tezacaftor and ivacaftor led to

MAC Lung Infections Best Treated When Specific Bacterial Species Known, Study Finds

Pulmonary infection with a specific species of the Mycobacterium avium complex (MAC), called Mycobacterium intracellulare, can be aggressive in people with cystic fibrosis (CF), with a greater loss of lung function evident but a lesser incidence of a secondary lung infection, a single-site study reports. This species also responded best to antibiotic treatment, its researchers said, suggesting that identifying “MAC isolates to the species level would have bearing on the prognosis and management” of these infections in CF patients. The study, “Distribution and outcomes of infection of Mycobacterium avium complex species in cystic fibrosis,” was published in the Journal of Cystic Fibrosis. MAC

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