News

CFF Awards Up to $2M to Advance Eloxx’s Therapy ELX-02

The Cystic Fibrosis Foundation (CFF) is awarding Eloxx Pharmaceuticals up to $2 million in additional funding to support the clinical program of ELX-02, a potential therapy for cystic fibrosis (CF) caused by nonsense mutations. “The Cystic Fibrosis Foundation is committed to accelerating treatments for the underlying cause of…

UK Rare Diseases Framework Opens to Improve Diagnosis, Access to Care

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…

Cholesterol Metabolism Impaired in CF Mouse Model

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…

Inhaled-IV Antibiotic Combo May Better Treat Acute Flares Than IV-only Mix

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…

Health Canada Giving Priority Review to Trikafta

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…

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