Two cystic fibrosis (CF) patients being treated for lung infections related to Mycobacterium abscessus — a multidrug-resistant nontuberculous mycobacteria (NTM) — using inhaled granulocyte-macrophage colony stimulating factor (GM-CSF) now both show signs of cure, Savara Pharmaceuticals announced. M. abscessus accounts for about 35 percent of NTM infections in cystic fibrosis patients in North America, researchers…
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Antimicrobial peptides administered as tiny nanoparticles show promise in treating bacterial infections without risking making the bacteria more resistant to treatment — as can happen with antibiotics, an EU-supported study reports. Unlike antibiotics, antimicrobial peptides or AMPs are part of the innate immune system, naturally occurring molecules that work as…
Disease severity in cystic fibrosis (CF) may be associated with an overreaction of the immune system to the fungus Aspergillus fumigatus, particularly due to a type of white blood cell called a phagocyte — which ingests and kills invading organisms — a study suggests. U.K.
A special formulation of an antioxidant-enriched multivitamin helped decrease the frequency of pulmonary exacerbations in patients with cystic fibrosis (CF), according to a new study. The findings, by researchers at Children’s Hospital Colorado and the University of Colorado School of Medicine, were published in the American Journal of…
New data suggest that inhalation of nitric oxide (NO) may improve lung function and alleviate the burden of serious lung infections, such as Mycobacterium abscessus complex (MABSC) and Pseudomonas aeruginosa, two serious pathogens affecting cystic fibrosis (CF) patients. Results from the Advanced Inhalation Therapies (AIT) pilot study were shared recently at the…
Arrowhead Pharmaceuticals’ investigational cystic fibrosis treatment candidate ARO-ENaC can effectively target the epithelial sodium channel alpha subunit (αENaC) in the lungs of rats, preclinical studies show. Given that ENaC is a well-known mediator of common cystic fibrosis symptoms, such as airway dehydration and mucus accumulation, these…
Celtaxsys has completed its Phase 2b trial assessing the safety and effectiveness of acebilustat (formerly CTX-4430) for cystic fibrosis (CF), with the company announcing the last visit of the last enrolled patient. Details of the trial, supported by the Cystic Fibrosis Foundation, will be presented at the upcoming 41st…
An antibacterial compound found in toothpaste, called triclosan, may be part of a future cystic fibrosis (CF) treatment, a Michigan State University study suggests. The research, “Triclosan is an aminoglycoside adjuvant for the eradication of Pseudomonas aeruginosa biofilms,” was published in the journal Antimicrobial Agents…
A baby born with cystic fibrosis (CF) in Germany today is likely to live to be 40 or 50, while another born in the Ukraine will probably die before the age of 18. That’s the appraisal of Hilde De Keyser, coordinator of Cystic Fibrosis Europe — a Brussels-based federation that…
The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…
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