News

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

A Phase 2 clinical trial investigating MS1819 in combination with stable pancreatic enzyme replacement therapy (PERT) to treat exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF) is actively recruiting participants in Europe, AzurRx BioPharma, the treatment’s developer, announced. The open-label study (NCT04302662) expects to enroll 24 CF patients, ages 12…

Ionis Pharmaceuticals’ investigational inhaled therapy IONIS-ENAC-2.5Rx showed promise in a Phase 1/2a study as a potential new treatment for cystic fibrosis (CF), the company announced, citing trial data. That clinical data showed that ENAC-2.5Rx led to a reduction in the expression of ENaC, a sodium transport…

Medicine by Design, which supports work in regenerative medicines, gave a 2020 New Ideas Awards to support a researcher exploring an alternative pathway to the CFTR protein as a possible treatment for cystic fibrosis (CF). The awardee, Amy Wong, PhD, works in developmental and stem cell biology at the…

A Phase 1b clinical trial evaluating Calithera Biosciences’ investigational oral therapy CB-280 is recruiting adults with cystic fibrosis (CF) and chronic airway infections at several U.S. sites. The trial’s design, along with CB-280’s preclinical data, was presented by Joel D. Mermis, MD, of the University…

Factors such as diabetes, a specific type of fungal infection, and previous mild to moderate episodes of hemoptysis, or coughing up blood, may help predict the risk of massive hemoptysis in people with cystic fibrosis (CF), a study suggests. The study, “Predictors of massive haemoptysis after…

Roche has acquired Enterprise Therapeutics’s TMEM16A potentiator portfolio, which includes ETD002, a therapeutic candidate being evaluated in Phase 1 clinical trials for the treatment of cystic fibrosis (CF). The TMEM16A portfolio is focused on treatments for CF and other severe respiratory disorders characterized by excessive mucus…

Exposure to tobacco smoke limits the medicinal benefit of Symdeko (tezacaftor/ivacaftor combo) in young patients with cystic fibrosis (CF), according to a recent study. This finding suggests that smoking must be avoided for Symdeko to provide its full benefits. It also suggests that the effects of smoke exposure…

A Phase 2 clinical trial of oral lenabasum in people with cystic fibrosis (CF) failed to meet its primary goal of reducing pulmonary exacerbations, the investigational therapy’s developer, Corbus Pharmaceuticals, announced. “We are very disappointed that the study did not meet the primary endpoint,” Barbara White, MD, chief medical officer and…

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…