U.K. Rare Diseases Framework, a government-led approach to raising national awareness of rare disorders, accelerating  diagnoses, and improving patient care and treatment, is now in practice. This  framework, signed by all four countries take make up the U.K. — England, Scotland, Wales and Northern Ireland — builds on the success…
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The metabolism of cholesterol is impaired in a mouse model of cystic fibrosis, a preliminary study suggests. In the model, there seems to be an altered production of bile salts (those that help with the digestion of fats) that may reduce the digestion and/or absorption of cholesterol. This increases cholesterol’s…
A Phase 1/2 clinical trial, called CYPHY, to evaluate the phage therapy YPT-01 in treating chronic Pseudomonas aeruginosa infections in people with cystic fibrosis (CF) has been initiated at Yale University. This double-blind, placebo-controlled study (NCT 04684641) aims to enroll 36 adult patients with these chronic infections. Additional…
The Cystic Fibrosis Foundation (CFF) encourages cystic fibrosis (CF) patients to speak to their healthcare team about COVID-19 vaccines, in agreement with recommendations from the U.S. Centers for Disease Control and Prevention (CDC). CFF will host…
Children with cystic fibrosis (CF) who live in more socioeconomically disadvantaged areas of the U.S. tend to have worse lung function and health outcomes than their counterparts in more affluent areas, a new study shows. The study, “The Association of Area Deprivation and State Child Health…
When given alongside the antibiotic Colistin (colistimethate), Cayston (aztreonam lysine), an inhaled antibiotic specifically targeting Pseudomonas aeruginosa, was superior to standard intravenous antibiotics at improving lung function and life quality among adults with cystic fibrosis (CF) and lung infections due to P. aeruginosa during an acute flare, a single-site…
Elevated levels of fecal calprotectin protein, a biomarker for intestinal inflammation, was linked to worse gastrointestinal symptoms and quality of life in children with cystic fibrosis (CF), a study reports. Measuring calprotectin may help clinicians better distinguish the source of gastrointestinal…
Health Canada is reviewing an application that seeks the approval of Trikafta, a triple combination reported to treat 90% of all with cystic fibrosis (CF), or those with least one F508del mutation. The request from Trikafta’s developer, Vertex Pharmaceuticals, covers eligible patients ages 12 and older, and has been granted…
Arcturus Therapeutics has selected ARCT-032, an aerosolized RNA-based therapy, to be developed as a novel therapeutic candidate for cystic fibrosis (CF). The therapy will use the company’s LUNAR delivery technology that comprises a library of more than 150 proprietary fatty molecules specifically designed to deliver RNA-based therapies…
Inhaled antibiotic tobramycin safely eradicated early Pseudomonas aeruginosa infection in cystic fibrosis (CF) patients younger than a year old, according to findings from a small retrospective study. The study, “…
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