News

AzurRx BioPharma has initiated a Phase 2 clinical trial testing its oral capsule MS1819-SD, combined with standard porcine pancreatic enzyme replacement therapy (PERT), in people with cystic fibrosis (CF) who have exocrine pancreatic insufficiency (EPI). PERT is the standard of care for EPI associated with…

People with cystic fibrosis (CF) may require a modified and more rigorous cleansing procedure to prepare their bowels for a colonoscopy screening procedure that allows good visualization, a study reports. Findings of the study, “A non-randomised single centre cohort study, comparing standard and modified bowel preparations, in adults with…

The diagnostic potential of breath analysis to detect serious lung infections in people with cystic fibrosis (CF) will be explored in a new clinical trial, which could lead to faster, more accurate diagnoses, and potentially improve the quality of life and prolong the life of these patients, researchers say. The…

Adding cystic-fibrosis-specific variables to the lung allocation score allows for more accurate identification of patients who are likely to benefit from a lung transplant, according to a new study. The study, titled “Effect of Including Important Clinical Variables on Accuracy of the Lung Allocation Score for Cystic…

The diagnostics company BillionToOne has launched its first product for commercial and clinical use — a blood test designed to help diagnose hereditary diseases in fetuses. The new product, called UNITY, tests cell-free fetal DNA to aid in the diagnosis of cystic fibrosis (CF), spinal muscular atrophy,…

AR-501, an investigational inhaled formulation of gallium citrate to treat chronic lung infections in patients with cystic fibrosis (CF), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The treatment, developed by Aridis Pharmaceuticals, is designed to be given once weekly…

Children often mistakenly pronounce cystic fibrosis (CF) as “65 roses.” Eilís Moroney and Ruth Cahill, both parents of children with this progressive lung disease, have turned that endearing practice into a book aimed at explaining CF to youngsters in an age-appropriate way. Titled “Our Baby has Sixty-Five Roses,”…

Ultrasound imaging can be a reliable diagnostic tool for early detection of liver disease in children with cystic fibrosis (CF), a study finds. Using this strategy can help clinicians identify CF patients most at risk for liver damage caused by cirrhosis (tissue scarring), researchers suggest. These findings were reported in…

More than 60 cystic fibrosis experts strongly support Relizorb being made available to CF patients on tube feeding, pushing in a commentary for the insurance coverage that’s generally denied this digestive enzyme cartridge, and inviting “the CF community” to become advocates of better patient access to the device. Their opinion…

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…