The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…
News
People with cystic fibrosis (CF) who were treated with Orkambi (lumacaftor/ivacaftor) have significantly less accumulation of fat in the liver — a condition known as hepatic steatosis — which is often associated with CF, a study showed. While hepatic steatosis is common in those…
Girls with cystic fibrosis (CF) under the age of six have higher levels of a tiny RNA molecule, miR-885-5p, in their blood compared with boys of the same age, which could be linked to the worse clinical outcomes normally seen in women with CF, a study suggests. The study,…
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.
Developing a way to destroy persister cells — bacterial cells that enter a dormant phase and are able to resist antibiotics — is the goal of Mehmet Orman, a professor at the University of Houston. The cells can cause recurrent lung infections in cystic fibrosis (CF) patients. “If…
An imbalance in the microbes that populate the gut of infants with cystic fibrosis (CF) affects their growth throughout the first year of life. This finding was detailed in the study “Fecal dysbiosis in infants with cystic fibrosis is associated with early linear growth failure” published…
The genetic programming that causes embryonic cells to differentiate into lung cells was described for the first time in a study in mice. Researchers say that understanding the genetic changes necessary for lung cell development could allow for more effective use of stem cells in research and treatment. The study,…
Allergan has entered into a definite agreement to sell Zenpep (pancrelipase), a therapy for exocrine pancreatic insufficiency due to cystic fibrosis (CF) and other conditions. The agreement was reached alongside the currently ongoing regulatory approval process for AbbVie’s acquisition of Allergan, and is contingent upon approval of…
The Cystic Fibrosis Trust is expanding its network of cystic fibrosis (CF) centers in the United Kingdom (U.K.) to give more patients access to clinical trials. Nine CF centers were chosen to be part of the Trust’s Clinical Trials Accelerator Platform based on their interest in clinical trials…
Streptococcus pseudopneumoniae should be included in the panel of opportunistic bacteria causing pulmonary exacerbations among those with cystic fibrosis (CF), according to a small French study. The study, “Streptococcus pseudopneumoniae, an opportunistic pathogen in patients with cystic fibrosis,” was published in the Journal of…
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