News & Articles

Celtaxsys Announces Last Patient, Last Visit in Landmark CF Phase 2b Lung Function Preservation Tria

ATLANTA, May 17, 2018 (GLOBE NEWSWIRE) -- Celtaxsys, Inc., a clinical stage pharmaceutical development company focused on advancing treatments for patients with rare inflammatory diseases, today announced the last patient, last visit in its 200-patient acebilustat Phase 2b lung function preservation trial for the treatment of cystic fibrosis (CF). This trial has been carried out with the scientific and financial support of the CF Foundation. “Completing this trial represents a significant milestone for the development of our lead medicine, the company and the CF community. We are excited to explore once daily acebilustat’s potential to reduce excessive lung inflammation as a complement to a

Toothpaste ingredient may bust up cystic fibrosis biofilms

A common antibacterial substance in toothpaste may combat life-threatening diseases such as cystic fibrosis when combined with an with an FDA-approved drug, researchers report. “We think this can save lives…” Researchers have found that when triclosan, a substance that reduces or prevents bacteria from growing, combines with an antibiotic called tobramycin, it kills the cells that protect the CF bacteria, known as Pseudomonas aeruginosa, by up to 99.9 percent. CF is a common genetic disease with one in every 2,500 to 3,500 people diagnosed with it at an early age. It results in a thick mucus in the lungs, which becomes a magnet for bacteria. These bacteria are notoriously difficult to kill b

Gastrointestinal Cancers More Common in Adults with CF, Especially After Transplant, Study Finds

Patients with cystic fibrosis (CF) are at higher-than-average risk of developing gastrointestinal cancers, especially those who underwent a lung transplant, a new study shows. Transplant patients with CF were found in this retrospective study to have a five-times greater risk of gastrointestinal cancer than those who have not had a transplant, the researchers found, emphasizing a need for careful screening for small intestine and colon cancers particularly, but also for biliary tract and pancreatic cancers. The study, “Risk of gastrointestinal cancers in patients with cystic fibrosis: a systematic review and meta-analysis,” was published in the journal The Lancet Oncology. More effective the

Improving Antibiotic Strategies To Combat Respiratory Infections In Cystic Fibrosis Patients

Cystic fibrosis (CF) is an inherited, progressive disease caused by mutations in the cystic fibrosis transmembrane regulator (CFTR) leading to persistent lung infections. Production of thick, viscous mucus within the lungs prevents cilia from beating on normal epithelia, allowing bacterial species to become entrapped. Respiratory infections are the most common cause of morbidity and mortality in CF patients. Pathogens frequently associated with this disease are Pseudomonas aeruginosa, Staphylococcus aureus, and Haemophilus influenza, while other species less frequently encountered are Stenotrophomonas maltophilia, Burkholderia cepacia, and non-tuberculosis mycobacteria. P. aeruginosa remains

Proteostasis Announces Formation of Independent Steering Committee of Leading Experts to Advise on t

Program to Pursue Broad Label for PTI-428 as an Add-On Therapy to CFTR Modulator Based Treatment BRIGHTON, Mass., May 8, 2018 /PRNewswire/ -- Proteostasis Therapeutics, Inc. (NASDAQ: PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, announced today the formation of an independent Steering Committee to guide the Phase 3 global clinical development program for PTI-428. This Steering Committee of leading CF experts will advise on the design and implementation of the Phase 3 global clinical program for PTI-428, the Company's novel cyst

Data from Spyryx Biosciences' HOPE-1 Phase 2 Clinical Trial to be Presented in a Late-Breaking O

DURHAM, N.C., May 8, 2018 /PRNewswire/ -- Spyryx Biosciences, Inc., a clinical-stage biopharmaceutical company developing novel therapeutics for severe pulmonary diseases, will present data from the first cohort of its ongoing Phase 2 clinical trial, HOPE-1, in a late-breaking oral presentation at the 41st European Cystic Fibrosis Conference. The data will be presented by Isabelle Fajac, M.D., Ph.D., Principal Investigator, Vice President and President Elect of the European Cystic Fibrosis Society, on June 8 during the conference in Belgrade, Serbia. HOPE-1 (Hydration for Optimal Pulmonary Effectiveness) is a randomized, controlled, 28-day Phase 2 study of Spyryx's lead therapeutic candidate

CFF ‘Venture Philanthropy’ Model Crucial to CF Breakthroughs and Group’s Success, CEO Says

When the Cystic Fibrosis Foundation (CFF) was established in 1955, most people with cystic fibrosis (CF) didn’t make it to their sixth birthday. Today, the average life expectancy of a CF patient is 47 years. To date, the U.S. Food and Drug Administration has approved 12 CF therapies. Three of them are CFTR modulators that treat the basic disease-causing defect, benefiting 60 percent of all patients, and more therapies are on the way. Preston W. Campbell III, the CFF’s president and CEO, directly attributes this dramatic improvement to the foundation’s philosophy of “venture philanthropy.” “We are now in Phase 3 CFTR trials that, if successful, will mean that as early as next year, more than

Carlow Cystic Fibrosis sufferer welcoming news that Orkambi will be available for children

A local Cystic Fibrosis sufferer says news that the drug Orkambi will now be available to children is fantastic. The Department of Health has announced that patients aged from six to 11 can now access the life-changing medicine. It means Ireland becomes one of the first countries in the EU to provide the drug to this age category. 21-year-old Carlow woman, Kelsey Nolan, first got to take the drug last year and she says it’s changed her quality of life hugely. Speaking to KCLR News, Kelsey says this is an extremely positive development and the drug has made such a huge difference to both her life and the life of her little brother, who has been taking it for six months now.

Myoepithelial Cells Are Reserve Stem Cells For Lung Airway Repair

A new population of stem cells that appear to be important for regenerating the airway following severe injury has been identified by researchers at the University of Iowa. The cells, known as glandular myoepithelial cells (MECs), were found to be surprisingly flexible. Our airway is a system of branching tubes that connects the nose and mouth with the lungs and allows us to inhale air, extract the vital oxygen, and exhale the waste product carbon dioxide. A layer of epithelial cells helps protect airways from harmful materials in the air we breathe. However, the cells that make up this first line of defense are vulnerable to damage and rely on local stem cells to repair and renew the barrie

Vertex Announces VX-445 Phase 3 Trial Program, Positive Data on VX-561 Combo Regimen

Vertex Pharmaceuticals is launching Phase 3 clinical trials to evaluate the safety and effectiveness of its investigational therapy, VX-445, in combination with VX-661(tezacaftor) and Kalydeco (ivacaftor) for cystic fibrosis (CF). The AURORA program will consist of two randomized, double-blind Phase 3 trials — AURORA F/MF and F/F trials — to test the triple combo therapy in patients with one or two copies of the F508del mutation in the CFTR gene — the gene that is defective in CF. The AURORA F/MF study is expected to enroll 360 participants, ages 12 and older, with one F508del mutation and one minimal function mutation in CFTR, who have not responded to VX-661, Kalydeco, or their combo regim

Galapagos to Start Trial Testing New Triple-Combo Therapy for CF

Galapagos is starting a clinical trial called FALCON to evaluate a new triple-combination therapy to treat cystic fibrosis patients. The combination treatment, using experimental therapies GLPG2451, GLPG2222, and GLPG2737, all developed by Galapagos, shows early potential in non-human studies for treating cystic fibrosis. In vitro assays have consistently shown that the triple-combination therapy has a significantly positive effect on human bronchial epithelial cells of CF with the F508del mutation, which is the most common mutation in CF patients. The in vitro results show that the therapy can significantly increase chloride transport, which is impaired in CF, compared to the current availa

True Health Launches New Genetic Carrier Screening

FRISCO, Texas, April 16, 2018 /PRNewswire/ -- True Health announced today that it will introduce new testing options for patients planning for their future families. The planTrue™ carrier screening from True Health employs industry-leading next-generation sequencing technology to detect whether a patient is a carrier for conditions such as cystic fibrosis, spinal muscular atrophy, fragile X, and many others. The American College of Obstetrics and Gynecology recommends that all women who are pregnant or considering pregnancy be offered carrier testing for cystic fibrosis and spinal muscular atrophy. Additional testing should be considered based on medical necessity in view of the patient's he

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