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Amikacin liposome inhalation suspension (ALIS) showed the same efficacy and similar safety profile as tobramycin inhalation solution (TIS) for the treatment of cystic fibrosis (CF) patients with Pseudomonas aeruginosa lung infection, a comparative clinical study showed. The study, “Amikacin liposome inhalation suspension for chronic Pseudomonas aeruginosa infection in cystic fibrosis,” was…

Claire’s Place Foundation is preparing for its 5th Annual Glow Ride event to raise money to support families affected by cystic fibrosis. The 3.4 mile-long bike ride will take place Saturday at 5:30 p.m. at the piers of Hermosa Beach and Manhattan Beach in California. In addition…

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…

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.