Treatment with ELX-02 in combination with Kalydeco (ivacaftor) was well-tolerated, but failed to meet efficacy goals in a clinical trial that tested the combo in people with cystic fibrosis (CF) caused by nonsense mutations. Eloxx Pharmaceuticals, the company developing ELX-02, says the lack of efficacy may…
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Acne might be a side effect of Trikafta therapy in cystic fibrosis (CF) patients, according to a new case series in the U.S. Trikafta (elexacaftor, tezacaftor, ivacaftor) is a CFTR (cystic fibrosis transmembrane conductance regulator) modulator that helps the defective CFTR protein function more effectively in CF…
Use of Kaftrio (elexacaftor, tezacaftor, and ivacaftor), sold in the U.S. as Trikafta, helped a woman with cystic fibrosis (CF) more easily become pregnant and maintain her health throughout the pregnancy and child’s birth, according to a case report from France. The woman had struggled in a previous pregnancy, due to…
Supplemental oxygen lessened the abundance of some microbial species in sputum samples from people with cystic fibrosis (CF) while sparing others, including Pseudomonas aeruginosa and Staphylococcus aureus, the main causes of bacterial lung infections in patients, a study reported. A need for caution exists when using supplemental oxygen, “historically ……
People with cystic fibrosis (CF) who have abnormal glucose tolerance — higher-than-normal blood sugar levels after eating — are more likely to experience clinical exacerbations of worsening lung function, according to a new study. This was found to be true even for patients who don’t meet the criteria for…
First Wave BioPharma has developed a new optimized formulation of adrulipase, a yeast-derived enzyme designed to treat exocrine pancreatic insufficiency in people with cystic fibrosis (CF). The new formulation ensures that the enzyme is packed in acid-resistant micro-granules and protected from the acid environment of the stomach. The enzyme then reaches…
In children with cystic fibrosis (CF), certain genetic variants were found to be associated with the rate of absorption of the antibacterial agent ciprofloxacin — used to prevent bacterial infections, mainly those caused by Pseudomonas aeruginosa — according to a new study. Researchers believe that these new data will…
The Cystic Fibrosis Foundation (CFF) has joined the CEO Action for Diversity and Inclusion, adding its name to the more than 2,300 corporate leaders, representing 85 industries, who have pledged to bring greater inclusion and diversity into their workplaces. Organizations with diverse teams perform better, the CFF stated…
The U.S. Food and Drug Administration (FDA) approved the expanded use of Orkambi (lumacaftor/ivacaftor) to treat children starting at 12 months old with cystic fibrosis (CF) who have two copies of the F508del mutation in the cystic…
A poorer adherence to pulmonary therapies does not seem to greatly influence disease severity in patients with cystic fibrosis (CF) who have a nearly normal lung function and good medication adherence rates overall, a study suggests. For this subgroup of patients, CF severity seems to not be associated with…
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