RCT223 and RTX0001, ReCode Therapeutics’ experimental RNA-based therapies for cystic fibrosis (CF), safely restored function to CFTR, the faulty protein in CF, in patient-derived lung cells. Delivered through the company’s non-viral platform — called the selective organ targeting (SORT) lipid nanoparticle (LNP) platform — the therapies were also…
News
Children and adolescents on Symdeko (tezacaftor/ivacaftor) and Orkambi (ivacaftor/lumacaftor) for cystic fibrosis (CF) had higher treatment adherence rates compared with adults given the same CFTR modulators, a nationwide study in the U.S. found. The study also showed that adherence — patients choosing to follow through with a prescribed treatment —…
Two months of treatment with Kalydeco (ivacaftor) by cystic fibrosis (CF) patients who carry a CFTR gating mutation significantly increased the diversity of bacteria in the gut but not in the respiratory system, a small study reported. A…
The U.S. Food and Drug Administration (FDA) has approved expanding the use of Trikafta (elexacaftor, tezacaftor, and ivacaftor) to children with cystic fibrosis (CF), ages 6 and older, who have at least one F508del mutation in the CFTR gene or a CFTR mutation that responds to Trikafta in laboratory studies. Use of…
Lower levels of certain volatile molecules in the breath of children and adults with cystic fibrosis (CF) can distinguish with good accuracy between those with and without Pseudomonas aeruginosa lung infection, a Dutch study shows. Notably, a reduction in the levels of a single molecule, called ethyl acetate, was…
A combination of bacteria-infecting viruses and antibiotics can be used to effectively protect lung cells against infection by Pseudomonas aeruginosa, a bacteria that commonly causes lung infections in people with cystic fibrosis (CF), according to a study led by researchers at the University of Geneva (UNIGE). The model…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
The nonprofit Cystic Fibrosis Canada has teamed up with technology company iMD Health Global (iMD) to make user-friendly digital health tools for cystic fibrosis (CF) available to both patients and healthcare professionals. The goal is to boost understanding of the disease and how to best manage it. Through…
A first-of-its-kind, observational study will allow patients with cystic fibrosis (CF) to use their smartphones to monitor the ways in which Trikafta (elexacaftor/tezacaftor/ivacaftor) affects their daily lives. Sponsored by the Cystic Fibrosis Foundation, the now-enrolling HERO-2 study (NCT04798014) will collect patients’ self-reported feedback for one year…
The therapeutic benefits of the triple-combination medication Trikafta are mostly due to the effects of elexacaftor, a next-generation corrector of CFTR, the faulty protein in cystic fibrosis (CF), a study has found. These findings suggest that new combinations with elexacaftor may be even more effective than Trikafta. Trikafta,…
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