News

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…

The complex diagnostic journey of a girl with cystic fibrosis (CF) and three different disease-causing mutations was described in a report by scientists in Italy. “This case well illustrates possible pitfalls in the clinical and molecular diagnosis of CF,” the researchers wrote. Their report, “Things come…

A noninvasive imaging technique called elastography can detect the degree of liver fibrosis, or scarring, in children with cystic fibrosis (CF), according to a recent study. Combining the technique with other laboratory tests in clinical practice might allow for the early diagnosis and treatment of liver disease in these…

The Cystic Fibrosis Foundation (CFF) has awarded $1.6 million to support research focused on identifying biomarkers of chronic lung allograft dysfunction (CLAD) — a complication of lung transplants — in people with cystic fibrosis (CF). A total of eight grants have been awarded, which will each focus on…

Home monitoring with a mobile phone-linked spirometry device may provide an effective way of detecting pulmonary exacerbations — episodes of a sudden worsening of respiratory symptoms — in people with cystic fibrosis (CF), a pilot study suggests. The findings showed more pulmonary exacerbations were identified through home monitoring…

The first patient has been dosed in a Phase 1/2 clinical trial evaluating 4D-710, an investigational genetic medicine for adults with cystic fibrosis (CF), its maker, 4D Molecular Therapeutics (4DMT) has announced. “The dosing of the first patient in the 4D-710 Phase 1/2 clinical trial in cystic fibrosis…

A new online exercise and yoga program for children and adolescents living with cystic fibrosis (CF) is in development by Beam and CF Yogi, and will launch — with no costs to youth — in August on Beam’s virtual well-being platform. The program, created with the Johns…

A noninvasive measurement of lung function called lung clearance index (LCI) was associated with the degree of lung damage over time in children with cystic fibrosis (CF), according to a recent study. LCI could be used to monitor lung disease progression in children with CF and may help clinicians…

KIT2014, an experimental treatment that Kither Biotech is developing to treat cystic fibrosis (CF) and other lung diseases, was able to reduce inflammation, relax the airways, and improve the functionality of CFTR modulators in preclinical models, a study shows. Kither is planning to launch a Phase 1/2a clinical…

In children with cystic fibrosis (CF) and a first or new-onset Pseudomonas aeruginosa infection, antibiotics given intravenously are not more effective at eliminating the bacteria than inhaled antibiotics given alone or as combination therapy, a Turkish study suggests.  Intravenous (IV) treatment was also associated with more pulmonary exacerbations and poorer lung function after…