A new, more efficient method for delivering gene therapies to lung tissues — based on tiny engineered proteins, called peptides —is creating therapeutic opportunities for people with diseases like cystic fibrosis, chronic obstructive pulmonary disease or COPD, and asthma. The potential of these new delivery tools was described…
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Pseudomonas aeruginosa, a bacteria that commonly causes serious lung infections in people with cystic fibrosis (CF), reacts to other dangerous bacteria in ways that could be exploited to develop new antimicrobial treatments, early research has found. When Staphylococcus aureus is present, P. aeruginosa starts moving…
People with cystic fibrosis (CF) show a “high adherence” to CFTR modulators, but these newer medications are costly and could undermine compliance to treatment over time, according to a joint studies by AllianceRx Walgreens Prime and Duquesne University School of Pharmacy. These findings were presented…
The Regional Pharmaceutical Procurement Service in Northern Ireland and the National Health Service (NHS) Wales have reached an agreement with Vertex Pharmaceuticals that will allow people with cystic fibrosis (CF) who live there to access all of Vertex’s currently approved medications for the disease. The terms of the…
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…
Proteostasis Therapeutics (PTI) has completed patient enrollment in a Phase 2 clinical trial evaluating its cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination therapies in cystic fibrosis (CF) patients who have at least one copy of the F508del mutation in the CFTR gene (the defective gene in CF…
Latinos in the Caribbean with cystic fibrosis (CF) carry rare CFTR gene mutations not often seen in Latin or other CF populations elsewhere, a study led by physician-scientists at the University of California, San Francisco (UCSF) found. CF patients in Puerto Rico and the Dominican Republic have CFTR…
The bionic pancreas (BP), an automated system that controls blood sugar levels, may be useful in managing cystic fibrosis-related diabetes, a small study says. The study, “Automated glycemic control with the bionic pancreas in cystic fibrosis-related diabetes: A pilot study,” was published in the Journal of…
Eloxx Pharmaceuticals presented additional positive preclinical and clinical data for ELX-02, its lead treatment candidate for cystic fibrosis (CF), reinforcing the therapy’s safety and tolerability in humans and its ability to increase the levels of CFTR protein in preclinical models of the disease. Findings were presented in three posters and…
Preclinical data shows that Arrowhead Pharmaceuticals‘ investigational inhaled treatment for cystic fibrosis (CF), called ARO-ENaC, accelerates mucus clearance and preserves lung function while being safe for the kidneys in animal models. The data was shared recently at the 2019 North American Cystic Fibrosis Conference (NACFC) in Nashville,…
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