The investigational therapy MS1819 showed positive safety results in a Phase 2 clinical trial for the treatment of exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF), AzurRx BioPharma announced. In people with CF, EPI can occur when mucus builds up and blocks the ducts of the…
cystic fibrosis
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…
Kristen Mudrack, PhD, a chemistry professor at Milligan College in Tennessee, has launched a research initiative to encourage college students to produce and test antibiotics that target and destroy Pseudomonas aeruginosa, a bacteria highly resistant to antibiotics that often causes lung infections in patients with…
The Cystic Fibrosis Foundation (CFF) is giving Eloxx Pharmaceuticals up to $1.61 million to support its planned Phase 2 clinical trial program assessing the safety, tolerability, and chemical properties of ELX-02, Eloxx’s lead investigational compound to treat cystic fibrosis (CF) caused by nonsense, or stop, mutations. The program…
CFTR channel activity is controlled by a molecular process known as phosphorylation. Now, a study suggests that the levels of phosphorylation affect how well Kalydeco works in people with certain mutations in the CFTR gene. The study “VX-770-mediated potentiation of numerous human CFTR disease mutants is influenced by phosphorylation level” was…
Adults with cystic fibrosis (CF) are less active than they should be, although they have comparable activity levels to people without the genetic disorder, according to a recent review. The review also showed that there are not enough studies that objectively assess physical activity levels in CF. Therefore,…
The lung clearance index (LCI) is more sensitive to changes in lung function in patients with mild cystic fibrosis (CF) than conventional spirometry parameters, a study asserts. The findings were reported in the study, “Lung clearance index to detect the efficacy of Aztreonam lysine inhalation in…
How well Acceptance and Commitment Therapy (ACT), or tailored “talk” therapy that can be delivered via telemedicine, helps people with cystic fibrosis (CF) cope with mental health issues will be the focus of an upcoming study. Supported by a three-year, $960,000 grant from the Boomer Esiason Foundation, the study is…
Through a new partnership with the Cystic Fibrosis Foundation (CFF), California-based winery Tarpon Cellars will donate $1 to the organization’s Georgia chapter for each bottle of wine sold. The funds will help support research and treatment development for cystic fibrosis (CF), an inheritable…
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…
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