Treatment with Kaftrio (elexacaftor, tezacaftor, and ivacaftor), sold as Trikafta in the U.S., may save people with cystic fibrosis (CF) an average of 15 minutes each day of respiratory physiotherapy to help with breathing, a study has found. The study, “Perceived burden of respiratory physiotherapy in people with cystic fibrosis taking elexacaftor–tezacaftor–ivacaftor combination: a 1-year observational study,” was published in Therapeutic Advances in Respiratory Disease. In CF, mucus produced by the lungs is stickier than normal, making it more likely for patients to develop an infecti ..read more

A test of physical function called Glittre-ADL may be better than the standard six-minute walk test at identifying physical limitations in people with cystic fibrosis (CF), a study reports. “To our knowledge, this is the first study to evaluate the performance of CF patients on TGlittre and to compare it with their [6-minute walk distance],” scientists wrote in the study “Evaluating the contribution of the Glittre-ADL test in adults with cystic fibrosis,” published in Physiotherapy Research International. Despite the availability of CFTR modulator therapies and other improvements in CF care ..read more

In a recent study, scientists described the process through which they discovered GLPG2737, an investigational CFTR modulator considered by Galapagos and AbbVie to be a possible treatment for cystic fibrosis (CF). The molecule was found in early work to have an additive effect when combined with other types of CFTR modulators. It was evaluated in Phase 1 and 2 clinical trials in CF patients in combination with approved and experimental modulator therapies, but no current clinical trials appear to be active. The study, “Discovery of GLPG2737, a Potent Type 2 Corrector of CFTR for the Treatment ..read more

Treatment with Trikafta (ivacaftor/tezacaftor/elexacaftor) eases nose and sinus symptoms in eligible patients with cystic fibrosis (CF), and the improvement in nasal symptoms is accompanied by an increase in nasal nitric oxide (nNO), a biomarker. The findings suggest “nNO could be used as an objective, noninvasive outcome measure for CF sinonasal [nose-and-sinus] disease,” researchers wrote in “Elexacaftor/tezacaftor/ivacaftor improves nasal nitric oxide in patients with cystic fibrosis,” which was published in the Journal of Cystic Fibrosis. Trikafta is a combination therapy of three CFTR mo ..read more

At 31, I still love basketball, and I’ve been a New York Knicks fan for 25 years. For the first 21 years of my fandom, the franchise was a model of instability and getting in its own way. I try to approach my health in the exact opposite manner, which is to say that I do so with similar determination and stability that the Knicks have had for the last four years. As an adult with cystic fibrosis (CF), I find that maintaining stability with my health is often more of a mental battle than a physical one. Overcoming the anxiety and fear that come with living with this disease isn’t easy. In Janu ..read more

A five-year study led by scientists at Emory University in Georgia aims to identify new ways to improve quality of life for people with cystic fibrosis (CF). The study, which will be led by Dio Kavalieratos, PhD, director of research at the Emory Palliative Care Center, is funded by a $3.1 million grant from the National Heart, Lung, and Blood Institute, part of the National Institutes of Health. “Our goal is to develop a nuanced, person-centered understanding of cystic fibrosis symptoms,” Kavalieratos said in a university press release. CF is a genetic disorder characterized by abnormally th ..read more

A novel treatment strategy for cystic fibrosis (CF) that’s based on a cellular structure called the porosome showed promise in cell and animal models of CF caused by the common F508del mutation. In the cell model, this approach was more potent than tezacaftor and ivacaftor, which are CFTR modulators used in approved CF therapies, according to data presented by Porosome Therapeutics, the company developing the approach. Findings were showcased at the April summit of the American Physiological Society in Long Beach, California, in the poster “Cystic fibrosis rescued using a reprogrammed po ..read more

Highly effective CFTR modulators, which target the cause of cystic fibrosis (CF), may ease signs of low iron levels in some patients with the disease, but low iron levels may remain even with treatment with the medications, a study finds. The scientists also emphasized that people with CF who are not on modulators due to lack of a qualifying mutation, intolerance to treatment, or lack of availability are prone to iron deficiency. The study, “Association between CFTR modulators and changes in iron deficiency markers in cystic fibrosis,” was published in the Journal of Cystic Fibrosis by resear ..read more

Biopharma and biotech companies have launched more than 450 clinical trials into potential new treatments for cystic fibrosis (CF) since 2018, according to a report from Novotech, a contract research organization that specializes in helping drug developers to run trials. “There are ample opportunities presented by advancements in technology, personalized medicine, and the emergence of new therapies, all of which hold the promise of improving outcomes,” the report states, adding that “the future for individuals with CF is increasingly hopeful.” CF is caused by mutations in the gene that e ..read more

I’ve had so many surgeries that I’ve lost track. You’d think I’d be used to the routine by now, but I’m not. With each surgery comes more anxiety as my trauma stacks up. I was convinced it’d get easier as I underwent more procedures, but I’ve learned that each one presents unique challenges. Growing up with cystic fibrosis (CF) brings a plethora of health hiccups. But while the CF community often talks about the physical symptoms we face, the emotional impact of our disease doesn’t get as much attention, nor does it resolve overnight. My CF led to a lifesaving double-lung transplant six years ..read more