Kalydeco (ivacaftor) therapy, given before age 1, was shown to normalize sweat chloride levels, and improve growth and lung and pancreas function in two baby boys with cystic fibrosis (CF), a case study reported. According to the researchers, these results show that Kalydeco “seems to be safe and…
The Cystic Fibrosis Foundation has invested $1 million in Felix Biotechnology to support the company’s efforts to develop a phage-based therapy as an alternative to antibiotics in controlling Pseudomonas aeruginosa infections in people with cystic fibrosis (CF). “We are incredibly honored and excited to receive this support from the CF…
Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…
The nonprofit Emily’s Entourage has awarded $440,000 in funding to support two projects aimed at advancing cystic fibrosis (CF) research. “These grants reflect Emily’s Entourage’s unwavering commitment to advance lifesaving research to fill critical unmet needs and expeditiously pursue promising therapeutic opportunities for the CF community, especially those that…
Cystic Fibrosis Canada’s 2020 annual data report from the Canadian Cystic Fibrosis Registry has been released, and it shows several improvements in the health of people with cystic fibrosis (CF), and a limited impact of the COVID-19 pandemic on health outcomes. “The Canadian Cystic Fibrosis Registry has…
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…
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