News

A team of researchers from the Michigan State University (MSU) College of Human Medicine and Spectrum Health will use a $2.1 million grant from the National Institutes of Health (NIH) to advance preclinical research on a gene therapy for cystic fibrosis (CF). The four-year award was given to the…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

MRI scans may help to reveal subtle changes in certain areas of the brain among people with cystic fibrosis (CF), a study found. The affected areas are involved in mood and cognition, which were found to be altered relative to people without the genetic disease. “CF patients exhibited significant…

The U.K. Cystic Fibrosis Trust, in collaboration with the U.S. Cystic Fibrosis Foundation, is launching a new strategic research center (SRC) dedicated to studying gene editing as a potential treatment strategy for cystic fibrosis (CF). “Making gene editing a reality for people with CF will take the…

Although person-to-person transmission is possible, most cystic fibrosis (CF) patients infected with Mycobacterium avium complex (MAC) do not share similar strains, according to a study of patients at U.S. care centers. Researchers found high genetic similarity between isolates of MAC across some people followed at the same CF centers.

A unique interaction between CFTR — the protein defective in people with cystic fibrosis (CF) — and two pro-inflammatory proteins may explain why some CF patients develop liver disease, researchers reported. Their work suggests that the CFTR protein can act as an anchor, bringing certain proteins — including the pro-inflammatory…

The school closures, at-home confinements and other safety measures necessitated by the COVID-19 pandemic have caused less anxiety among children with cystic fibrosis (CF) than age-matched peers without the disease, a survey study in Turkey found. Children and teenagers with CF “seem to show more resilience in coping with…

Since the introduction of Kalydeco (ivacaftor) in the U.K., marked changes in treatment patterns for cystic fibrosis (CF) patients using the medication have become noticeable over time, a study reported. Specifically, people treated with Kalydeco were less likely to continue with other treatments, such as inhaled antibiotics or…

Bacteria living in the gut are abnormally slow to develop functional abilities in infants with cystic fibrosis (CF), which may affect their nutrition, growth, and gastrointestinal health, a study reported. Therapies targeting the gut microbiome could be “promising avenues” to improving their overall health, its researchers suggested. The study,…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…