News

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…

Twincer, a dry powder inhaler (DPI), was shown to be a more attractive approach for the inhalation of colistin — an antibiotic used to treat lung infections in people with cystic fibrosis (CF) — when compared with a nebulizer, according to a new study. The findings…

Konglomerate Games, the student-made company that developed a video game to encourage children with cystic fibrosis (CF) to do their breathing exercises, won first prize in the 2020 Santander Universities Entrepreneurship Awards. These awards are part of the Santander Universities Emerging Entrepreneurs Programme, which has been created to…

The frequency of nontuberculous mycobacteria (NTM) infections in children and adolescents with cystic fibrosis (CF) in the U.K. remained stable from 2016 to 2018, but substantially higher than at the start of the decade, according to a recent study. Data also showed that allergic bronchopulmonary aspergillosis (ABPA)…

Oral antibiotics are just as effective as intravenous (into-the-vein) antibiotics at killing Pseudomonas aeruginosa bacteria in people with cystic fibrosis (CF), a new study shows. This finding may allow people with CF to spend less time in the hospital to receive treatment, ultimately reducing treatment burden. The study,…

Tablet-based hearing tests can accurately screen for hearing loss in people with chronic lung diseases such as cystic fibrosis (CF) without specialist supervision, according to a recent study. This finding suggests that tablets may be an inexpensive and practical addition to programs that monitor patients for signs of hearing…

Spirovant Sciences‘ lead gene therapy candidate for select cystic fibrosis (CF) patients, SPIRO-2101, was given rare pediatric disease and orphan drug designations by the U.S. Food and Drug Administration (FDA) to support its development, according to a recent press release. Orphan drug designation applies to medicines targeting…

The U.S. Food and Drug Administration (FDA) has approved Vertex Pharmaceuticals‘ Kalydeco (ivacaftor) as a treatment for infants as young as four months with cystic fibrosis (CF) caused by certain mutations. The approval makes Kalydeco the first medication in its class to be approved for…