News

British researchers have come up with a new way to screen for compounds that could be used to treat inherited diseases such as cystic fibrosis and Duchenne muscular dystrophy. The approach also may work with gene therapies, whose aim is to overcome abnormalities stemming from defective genes. These abnormalities can cause…

A pancreas abnormality is a sign that young children with cystic fibrosis may develop diabetes later in life, a study reports. The abnormality is fewer cells that produce and regulate blood sugar. These beta cells are part of clusters of different cells that scientists call islets. Researchers published their work…

Researchers determined the structure of a calcium-activated chloride channel, called TMEM16A, which they suggest could offer a new way of treating cystic fibrosis (CF). The study by scientists at University of Zurich, Switzerland, reporting these findings is titled “Activation mechanism of the calcium-activated chloride channel TMEM16A…

One of the most frequently mutated genes in cancer, PTEN, in involved in the repeated lung infections that trouble cystic fibrosis patients — not because PTEN is abnormal in CF, but because it is unable to work with CFTR gene, which is mutated in this disease, to help clear the…

Preventing bacteria from communicating with each other may be a way to bolster the effectiveness of current cystic fibrosis (CF) therapies, a study reports. According to researchers at the University of Montreal Hospital Research Centre (CRCHUM), the use of quorum-sensing inhibitors can help to prevent the harmful effects of…

Mothers of young children with cystic fibrosis bear a heavier caregiving load than fathers, according to an Irish study. The research also showed that the caregiving burden increased as children grew older and when they became infected with the bacteria Pseudomonas aeruginosa. Although University College Dublin researchers identified circumstances that could affect…

Clinical trials on all three Proteostasis Therapeutics’ investigational cystic fibrosis modulators — PTI-428, PTI-801, and PTI-808 — have demonstrated they are safe and have the potential to improve the effectiveness of Orkambi in patients with cystic fibrosis (CF). The positive clinical data supports the initiation of new clinical trials evaluating the…

Jim Best, a 34-year-old Canadian pharmacist with cystic fibrosis, is also a talented photographer who rides his motorcycle across the country every year to raise funds for CF research. In addition to generating funds, the trips help the Sackville, Nova Scotia, resident spread awareness about the incurable disease that affects…

Arch Biopartners recently completed a good manufacturing practice (GMP) production campaign for AB569, a potential inhalation treatment for antibiotic-resistant bacterial lung infections in people with cystic fibrosis (CF) chronic obstructive pulmonary disease (COPD) and other conditions. The campaign, intended to ensure the quality of the investigative therapy, was…

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,…