cystic fibrosis

Health Canada has approved Orkambi (ivacaftor/lumacaftor) to treat preschoolers with cystic fibrosis (CF) who carry the F508del mutation in both gene copies of the CFTR gene. Orkambi, developed by Vertex Pharmaceuticals, in the first disease-modifying therapy in Canada for children ages 2 to 5 with CF. It previously was …

Aridis Pharmaceuticals has enrolled the first healthy participant in its Phase 1/2a clinical trial to evaluate the antibacterial potential of its investigational candidate, AR-501 (gallium citrate), against chronic lung infections in patients with cystic fibrosis (CF). The study (NCT03669614) is expected to enroll approximately 48 healthy adult volunteers and…

The effects of CFTR modulator therapies such as Kalydeco (ivacaftor) can be more reliably assessed using a test that specifically measures the sweating dependent on CFTR, called C-sweat rate, than using classic sweat tests or lung function measurements, a study reports. This kind of test was sensitive enough to reveal that…

Wrist-worn monitoring devices are an acceptable method of objectively tracking the levels of physical activity of children and young people with cystic fibrosis (CF), a study reports. The study, “A Formative Study Exploring Perceptions of Physical Activity and Physical Activity Monitoring Among Children and Young People with Cystic Fibrosis…

A recent study confirmed the presence of increased serum levels of interleukin-7 (IL-7) in cystic fibrosis (CF) patients, compared with healthy controls. The researchers could establish a link between higher IL-7 levels and worse lung function in patients, suggesting a possible biomarker for CF. The study, “Higher…

Air Next, a small, simple-to-use handheld spirometer, could transform cystic fibrosis (CF) treatment, allowing patients to monitor their own lung function at home and share the results remotely with their healthcare team. Air Next connects via Bluetooth to a user-friendly smartphone app called Aria. Both Air Next spirometer and the Aria…

Aridis Pharmaceuticals is planning to launch a Phase 1/2a clinical trial in the beginning of 2019 to evaluate the potential of its investigational antibacterial compound AR-501 (gallium citrate) in patients with cystic fibrosis (CF). This follows the approval of the Investigational New Drug application for AR-501 by…

Preschoolers with cystic fibrosis (CF) treated with inhaled hypertonic saline in a clinical trial showed significant improvement in lung function, PARI, the company whose solution and equipment was used in the study, reports. The Saline Hypertonic in Preschoolers (SHIP) study (NCT02378467) examined the effectiveness of treatment with 7% hypertonic saline…

It’s one of the ironies of cystic fibrosis (CF) conferences that the patients who most urgently need the information are discouraged from attending for fear of cross-contamination. Under infection control guidelines published by the Cystic Fibrosis Foundation (CFF), only one CF patient may be in a room at a…

For the second year in a row, Mastercard ranks as the largest corporate sponsor of the Greater New York chapter of the Cystic Fibrosis Foundation (CFF). This year alone, Mastercard has raised more than $90,000 from the Great Strides walk, and supported other CFF events such as the…