News

The Rare Disease Cures Accelerator-Data and Analytics Platform — dubbed RDCA–DAP — has received new datasets that promise to accelerate research and the development of new therapies for cystic fibrosis (CF). The data was generated by the Cystic Fibrosis Therapeutics Development Network (TDN), known for its clinical trials…

An infant boy, who was first seen with recurrent infections and a failure to thrive, was diagnosed with cystic fibrosis (CF) caused by a rare genetic mutation, a case study reports. “This case underscores the importance of considering CF even when classic symptoms are absent, as genetic mutation can…

A 3-year-old boy who carries a new combination of two mutations in the CFTR gene, but with no clinical signs of cystic fibrosis (CF), was described by researchers in a case report. “The case presented has no clinical symptoms so far and doesn’t meet the criteria for a…

The U.S. Food and Drug Administration (FDA) has cleared a Phase 2 clinical trial testing ARCT-032, an inhaled therapy candidate, in people with cystic fibrosis (CF). Arcturus Therapeutics, the therapy’s developer, did not announce when the trial is expected to start. “The study is designed to evaluate the…

A report described episodes of skin rash associated with joint pain and inflammation, known as dermatitis-arthritis syndrome, in four females with cystic fibrosis (CF). The patients had similar symptoms, including pink, swollen, itching skin eruptions associated with joint pain. The researchers proposed cystic fibrosis dermatitis arthritis syndrome as a…

Gene therapy was found to work as well as a combination of the three CFTR modulators in Trikafta to restore the function to the CFTR protein, which is faulty in cystic fibrosis (CF), with the potential to work for all patients, regardless of their disease-causing mutation. That’s according…

The real-world use of Kalydeco (ivacaftor) effectively eased disease severity in people with cystic fibrosis (CF) who had certain disease-causing mutations, according to a study that included patients with mutations for which Kalydeco was approved in the U.S. in 2017 based largely on laboratory data. “This study adds…

A new small molecule, called VOMG, showed good antimicrobial activity against Mycobacterium abscessus, a bacterium that can cause serious infections in people with cystic fibrosis (CF). “The bactericidal and eradicating properties of VOMG … strongly reinforce the potential of VOMG in the treatment of pulmonary infections especially in CF individuals,…

A high dose of vitamin D combined with inulin, a prebiotic, led to improvements in the diversity of bacteria living in the airway and lungs of people with cystic fibrosis (CF) in a small clinical trial. The findings in the study, “Impact of high-dose cholecalciferol (vitamin…

While physical health and life quality gains can be evident in people using CFTR modulators, a group of disease-modifying therapies for cystic fibrosis (CF), their mental well-being needs more attention, according to the Wellness in the Modulator Era (Well-ME) study. Nearly 1 in every 4 patients reported poorer…