News

Eloxx Pharmaceuticals presented new positive preclinical data for ELX-02, its lead treatment candidate for cystic fibrosis (CF), demonstrating that the therapy increases the levels of CFTR protein and its correct placement at the cell’s surface in patient-derived organoids. Findings were presented by Shira Landskroner-Eiger, PhD, Eloxx’s principal…

Costs for approved cystic fibrosis (CF) treatments and new approvals — including therapies extended to more age groups or targeting new mutations — could double in price, according to pharmacy benefit manager (PBM) Prime Therapeutics. The cost increases could result in higher premiums for Prime members. According to…

Treatment with Proteostasis Therapeutics’ triple combo therapy regimen may significantly improve the lung function of patients with cystic fibrosis (CF), results from a Phase 1 clinical trial suggest. The therapy includes the company’s three investigational modulators of the CFTR gene: PTI-428, which is an amplifier; PTI-801,…

Xolair (omalizumab), a medicine used for allergic asthma and chronic hives, is effective for treating lung allergic reactions caused by fungi in patients with cystic fibrosis (CF), a case report suggests. The study reports three cases of allergic bronchopulmonary…

Targeting some elements in the bacterial cell wall can be an effective way to activate the immune system and fight chronic, treatment-resistant Pseudomonas aeruginosa infections, according to a study. The finding was reported in a study titled “Profiling the susceptibility…

Removing a key metabolite called pyruvate from Pseudomonas aeruginosa and Staphylococcus aureus bacteria biofilms — two of the most common bacteria found in cystic fibrosis (CF) patients — could help boost the effectiveness of antibiotics, a study reports. The study, “Pyruvate-depleting conditions induce biofilm dispersion and enhance…

A new method to estimate the best dose of enzyme supplements for pancreatic enzyme replacement therapy (PERT) based on different meals shows promise in young patients with cystic fibrosis (CF), according to a pilot study. According to…

Amphotericin B, a widely used antifungal medication, may be a potential treatment to restore lung mucus properties, including its ability to fight infections, in people with cystic fibrosis (CF), a preliminary study shows. The medicine creates pores in the membrane of cells lining the airways, working as a…

Warning: Spoilers for the movie are contained in this story.  Mention a rare disease milestone — and a new approach to treatment, a new medicine, or even a cure comes to mind. March 15, though, marks a milestone of a different sort for cystic fibrosis (CF) patients: the debut of…

Cystic fibrosis (CF) patients in Australia age 12 and older, who have two copies of the F508del mutation in the CFTR gene (a copy inherited from each parent), now can be treated with Vertex Pharmaceuticals’ Symdeko (tezacaftor/ivacaftor and ivacaftor). The treatment also was approved for any mutation in the CFTR gene that responds to Symdeko based on lab…