News

Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…

Destiny Pharma has received an award from the Cystic Fibrosis Foundation (CFF) to evaluate its investigational compound, XF-73, as a potential treatment for methicillin-resistant Staphylococcus aureus (MRSA) lung infections in people with cystic fibrosis (CF). The study will be carried out by researchers affiliated with the CFF,…

Children with cystic fibrosis (CF) who have lung infections due to CF-related bacteria, especially Pseudomonas aeruginosa, were found to have higher levels in their bloodstream of biomarkers of oxidative stress and related tissue damage. These high levels were accompanied by reduced lung function and worse nutritional status, according…

The government of Ontario has expanded coverage of Vertex Pharmaceuticals’ triple-combination treatment Trikafta for children, 6 and older, with cystic fibrosis (CF) and at least one copy of the most common CF-causing mutation, called F508del. The announcement comes a few months after Health Canada expanded Trikafta’s previous label,…

A technique called 2D shear wave elastography, which measures the stiffness of tissues, could be useful for detecting liver disease in people with cystic fibrosis. That’s according to the study, “2D Shear Wave Elastography, a promising screening tool for Cystic Fibrosis liver disease, shows a correlation…

Most adults with cystic fibrosis (CF) report experiences with painful or frightening medical procedures in childhood after their diagnosis, according to a U.K. study. These patients may benefit from psychosocial support from CF teams, who can ask about trauma in childhood in a safe and sensitive manner, researchers say.

Glatiramer acetate, an approved multiple sclerosis (MS) therapy, improved the effectiveness of the antibiotic tobramycin against Pseudomonas aeruginosa, a type of bacteria that commonly infects the lungs of people with cystic fibrosis (CF), a study demonstrated. The findings support glatiramer acetate as a promising antibiotic add-on therapy candidate,…

Researchers have developed a way to monitor the onset and progression of lung disease in young children with cystic fibrosis (CF) for whom lung imaging and diagnostic techniques are not well established. Testing the approach in 145 CF infants, the researchers found that about half showed signs of early-onset…

Six months of Kaftrio (elexacaftor-tezacaftor-ivacaftor), which is marketed as Trikafta in the U.S., led to marked reductions in abdominal symptoms among people with cystic fibrosis (CF), a study in Germany and the U.K. found. “To our knowledge, this is the first study assessing gastrointestinal symptoms, with a validated…

One year of Trikafta treatment for adults with cystic fibrosis (CF) post-lung transplant resulted in only limited improvements in sinus and digestive problems and was not well-tolerated, a small study found. In fact, among the 13 patients in the study, the discontinuation rate for the triple-combination therapy was 38.4%.