Blocking a pro-inflammatory protein complex, broadly known as the neutrophil inflammasome, represents a new way of treating chronic Pseudomonas aeruginosa infections in cystic fibrosis (CF) patients, a proof-of-concept study demonstrated. The study, “Specific Inhibition of the…
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Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…
Clinical centers with high screening rates for cystic fibrosis-related diabetes (CFRD) were more likely to achieve early CFRD diagnosis and prevent lung function decline, a study has found. This was reported in a study, “The association of pediatric cystic fibrosis-related…
The presence of the fungus Aspergillus fumigatus is linked to lower respiratory-related quality of life in people with cystic fibrosis (CF), a study has found. The study, “The presence of Aspergillus fumigatus is associated with worse respiratory quality of life in cystic fibrosis,” was published in…
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…
A young girl with cystic fibrosis (CF) and acute recurrent pancreatitis was able to eat some food normally and experienced relief of her abdominal symptoms after undergoing a complete removal of her pancreas and a transplant of pancreatic cells into her liver, according to a case report. At…
Pulmonary infection with a specific species of the Mycobacterium avium complex (MAC), called Mycobacterium intracellulare, can be aggressive in people with cystic fibrosis (CF), with a greater loss of lung function evident but a lesser incidence of a secondary lung infection, a single-site study reports. This species also responded best…
The U.S. Food and Drug Administration (FDA) has granted priority review to Vertex’s new drug application for its triple combination — elexacaftor (VX-445) plus tezacaftor and Kalydeco (ivacaftor) — for cystic fibrosis (CF) patients who cannot use the company’s other disease-modifying treatments or don’t benefit as intended.
Path BioAnalytics (PBA), a biotechnology company based in North Carolina, has licensed the rights to further develop cavosonstat (N91115), a targeted therapy for cystic fibrosis (CF), from the Chinese company Laurel Therapeutics. Cavosonstat is a small molecule designed to increase the stability of the CFTR protein, defects of which cause CF.
A defect in cellular metabolism is the root cause for the high incidence of lung infections caused by the bacteria Pseudomonas aeruginosa in patients with cystic fibrosis (CF), a study has found. Results from the study, “CFTR-PTEN–dependent mitochondrial metabolic dysfunction promotes Pseudomonas aeruginosa airway infection,” were published in…
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