Kalydeco (ivacaftor) is safe and effective in 1- and 2-year-olds with cystic fibrosis (CF), Vertex Pharmaceuticals reported based on data from its ongoing Phase 3 clinical trial in infants and toddlers. The treatment’s safety was similar to that seen in studies of older children, the company said. Moreover,…
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Doctors should frequently re-evaluate the use of protein pump inhibitors (PPIs) for cystic fibrosis (CF) patients, urges a University of Florida study which warns that long-term PPI use leads to a higher risk of hospitalization for pulmonary exacerbations. Identifying risk factors associated with pulmonary exacerbations is critical since they cause a decline…
Canadian researchers developed a safer and more cost-effective method for transforming differentiated adult cells into stem-like cells, a process known as induced pluripotent stem cells (iPS). The new method, called interrupted reprogramming, already showed promise as a potential cell therapy replacement in a mouse model with cystic fibrosis. The study “…
The Colton Underwood Legacy Foundation has partnered with International Biophysics, the manufacturer of AffloVest, to provide 50 cystic fibrosis (CF) patients with the next-gen airway clearance therapy in all 50 U.S. states. The foundation, created in 2015 by former NFL linebacker Colton Underwood, encourages youth participation in…
The Cystic Fibrosis Foundation Therapeutics (CFFT) has given an award worth up to $5 million to Savara Pharmaceuticals to support the continued development of AeroVanc, an inhaled antibiotic intended to treat persistent methicillin-resistant Staphylococcus aureus (MRSA) lung infection in individuals with cystic fibrosis (CF). CFFT is the non-profit drug discovery…
Sevion Therapeutics and Eloxx Pharmaceuticals announced that a first healthy subject has been dosed in a Phase 1b clinical trial assessing the safety, tolerability and drug properties of ELX-02 as a potential treatment of several genetic diseases caused by nonsense mutations, including cystic fibrosis (CF). Nonsense mutations are…
Michelle Laver remembers putting a rattle in her infant daughter’s hand and closing her little fingers around it. Then she watched the rattle fall. Kate couldn’t hold on to it. Michelle and Richard Laver, founders of Kate Farms, with Kate, 11. (Photo courtesy of Kate Farms) Kate eventually…
AIT Therapeutics’ inhaled nitric oxide formulation improved the breathing and physical functioning of cystic fibrosis patients with a lung infection, a Phase 2 clinical trial shows. The preliminary results applied to the formulation’s use against nontuberculous mycobacteria, which are collectively known as the mycobacterium abscessus complex. AIT said the NO-NTM…
Genetic screening for cystic fibrosis mutations helps individuals and couples identify their risk of having a child with the disease, an Australian study shows. The research, published in the journal Genetics in Medicine, is titled “Reproductive genetic carrier screening for cystic fibrosis, fragile X syndrome, and spinal…
A recent study found improvements in lung function and body mass index (BMI) among children and young adults with cystic fibrosis (CF) both in the United States and Canada from 1990 to 2013, though the American patients improved faster than those in Canada. The study, “Comparison of Nutrition and…
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