Orkambi (lumacaftor/ivacaftor), a treatment for cystic fibrosis (CF) patients in Australia age 6 and up, is closer to being subsidized by the government, according to Vertex Pharmaceuticals, the medication’s manufacturer. The company received a recommendation from the Pharmaceutical Benefits Advisory Committee (PBAC), an independent expert body, to list…
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When Shira Zagury joined the Cystic Fibrosis Foundation of Israel as CEO eight years ago, only one cystic fibrosis (CF) patient sat on the nine-member board of directors. Today, five of the nine are adults with CF — a reflection of the ever-increasing life expectancy of those born with the disease.
The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) as the first-ever therapy to treat the underlying cause of cystic fibrosis (CF) in 1-year-old toddlers, Vertex Pharmaceuticals announced. “Parents and physicians now have a medicine to treat the underlying cause of CF in patients as young as one…
A study in mice showed it is possible to transplant stem cells into the lungs and replenish the airways with healthy cells, an approach that could combat cystic fibrosis and alleviate its symptoms. Researchers used stem cells from the airways of cystic fibrosis (CF) patients, which were corrected to produce…
In addition to mutations in the CFTR gene, cystic fibrosis (CF) patients with pancreatitis also have a high prevalence of mutations in genes regulating pancreatic function, according to researchers. Conducted by a research team in Italy, the study, “Trans-heterozygosity for mutations enhances the risk of recurrent/chronic pancreatitis…
Patients with cystic fibrosis (CF) have a greater risk of developing abnormalities in their retinal veins, which can impair visual acuity, a case report suggests. But researchers claim the higher risk is not due to one single factor, but to several contributing risk factors for vascular events. The study,…
A clinical study into high-frequency chest wall oscillation vests — assessing their short-term impact on standard measures of lung function before and during use — challenges the view that these devices work through airflow bias in the lungs, the process responsible for mucus movement when breathing. Findings, using established tests that include forced vital capacity…
The active ingredient of Synspira’s SNSP113, an inhaled investigational treatment for chronic lung infections, demonstrated an ability to completely eliminate antibiotic-resistant Pseudomonas aeruginosa bacteria, a major cause of lung disease in…
Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor), a licensed medicine used to treat cystic fibrosis (CF) patients ages 6 and older, has now been approved by the U.S. Food and Drug Administration as the first medicine to treat CF in children ages 2-5. The approval is for children who have…
Mologic, an U.K.-based company developing personalized diagnostic devices, announced the beginning of a Phase 1/2 clinical trial to test its urine-based diagnostic tool, HeadsUp, to monitor pulmonary exacerbations in patients with cystic fibrosis (CF). The study is designed to identify biomarkers, present in urine of CF adult patients, that…
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