News

Bionews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A…

Multiple breath washout testing is superior to chest X-rays at detecting early signs of cystic fibrosis (CF) in infants and preschoolers, a study shows. The data also suggest that combining information from both techniques provides a considerable degree of assurance regarding the absence or potential presence of early lung…

The Cystic Fibrosis Foundation’s 2022 CFF Impact Grant program is now accepting applications from individuals or nonprofit groups seeking funding for projects that support cystic fibrosis patients or their families in their everyday lives. Those wishing to develop such efforts — particularly virtual support programs — also may…

A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…

Treatment with Trikafta is linked with fewer infection-related hospitalizations and a reduced need for antibiotics among people with cystic fibrosis, a study based on real-world U.S. data indicated. The study, “The rapid reduction of infection-related visits and antibiotic use among people with cystic fibrosis…

For people with cystic fibrosis (CF) who are not eligible for treatment with a CFTR modulator, seeing other patients benefit from these new therapies is “both uplifting and disheartening,” according to a new study. “Although most are happy for those who are benefiting from modulators, they are eager…

Most people with cystic fibrosis (CF) responding to a U.S.-based online survey were generally satisfied with their treatment plan and were not considering changing their medication. The survey was conducted by Cystic Fibrosis News Today. Shortness of breath was the most commonly reported symptom among CF patients, as well as…

The Canadian Institutes of Health Research (CIHR) has awarded CA$810,000 (about $637,000) over five years to researchers at the University of Saskatchewan (USask) to support their research exploring the role of two cell types in the lungs of patients with cystic fibrosis (CF). A better understanding…

The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…

Worldwide, only 12% of people with cystic fibrosis (CF) have access to the triple combination therapy Trikafta, despite this next-generation medicine being suited to a “large proportion” of patients, a study on global rates of CF diagnosis and treatment reported. Global estimates of total CF patients are also likely underestimated due…