News

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

For the first time, bacteriophage therapy treated a boy with chronic and antibiotic-resistant Achromobacter bacteria infection following a double lung transplant due to cystic fibrosis (CF), a case study reported.  The study, “…

The Achromobacter bacteria species that is an increasing source of chronic lung infections in people with cystic fibrosis (CF) shows evidence of patient-to-patient transmission and emerging antibiotic resistance, scientists in Denmark report. Their study, “…

Hospitalized children and adolescents with cystic fibrosis (CF) who can take enzyme treatments for pancreatic insufficiency on their own, rather than waiting for them to be given by a nurse, are more likely to take these necessary medications at the proper time with less of a wait, a survey…

Two new sites for the extension of a Phase 2b trial testing an immediate release capsule form of MS1819 in treating exocrine pancreatic insufficiency in cystic fibrosis (CF) have opened in Poland and are enrolling patients by invitation. Part of the OPTION 2 study (NCT04375878), these European sites join those already open in…

The Cystic Fibrosis Foundation (CFF) has given Kinnear Pharmaceuticals up to $3 million to conduct the laboratory tests of CSA-131, a potential inhaled treatment of infections in people with cystic fibrosis (CF), that are needed to move it into human trials. CF patients are prone to bacterial lung…

The U.S. Food and Drug Administration (FDA) has accepted for review an application to expand the use of Trikafta as a treatment in children with cystic fibrosis (CF) ages 6 to 11 with at least one F508del mutation in the CFTR gene (the gene defective in…

Using bronchoscopy to examine the airways and the surrounding tissues of people with cystic fibrosis, coupled with the collecting of lung fluid through bronchoalveolar lavage (BAL), is safe and a reasonable alternative when sputum samples are not possible, a study reports. Adult patients participating in research reported mild side effects…

Cystic fibrosis (CF) patients and carriers — those with mutations in only one copy of the CFTR, the gene associated with CF — of European ancestry are at a higher risk of developing pneumonia, according to a U.S. study of genetic risk for this lung disease. CF patients were also…

Individuals with cystic fibrosis (CF) from racial and ethnic minority groups in the U.S. are twice as likely to be ineligible for disease-modifying therapies than white patients, because their mutations are not known to be suited to approved treatments, a study reports. The resulting lack of treatment access exacerbates…