cystic fibrosis

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.

Should scientists have the right to edit the genes of future generations to eliminate hundreds, if not thousands, of potential rare diseases? Or should researchers restrict their use of genome editing to somatic cells, so that they don’t pass changes on to the next generation? What about stem-cell research, three-parent…

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…

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…

Two years after approving it, the 28-member European Union will begin enforcing its General Data Protection Regulation (GDPR) — a tough new law that aims to protect the EU’s 512 million citizens, including rare disease patients, from having their medical records misused, sold, or subject to extortion by hackers, third…

Vast Therapeutics, formerly known as Novoclem Therapeutics, recently announced that the company’s first controlled nitric oxide (NO) release powder-based therapy candidate, BIOC51, can substantially reduce the bacterial load of nontuberculous mycobacteria (NTM) in the lungs of animal models of cystic fibrosis. Vast Therapeutics is a preclinical stage pharmaceutical…

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…