A cystic fibrosis expert will be among more than 80 speakers at this month’s Rare Disease & Orphan Products Breakthrough Summit in Washington. The nonprofit National Organization for Rare Disorders (NORD) will be hosting the sixth annual event Oct. 16-17 at the Marriott Wardman Park Hotel. It is expected to attract…
Researchers have discovered that the Bpifb1 gene plays an important role in controlling levels of a mucin protein called MUC5B, one of the main components of mucus. This means that the gene also plays a crucial role in protecting the body’s airways. The team found that deleting the Bpifb1 gene in…
Women with diabetes associated with cystic fibrosis (CF) do not risk their disease worsening by becoming pregnant. Their children also are not affected by the presence of diabetes, according to a study by researchers at the University of Lyon in France. The only significant difference the study found between…
Preclinical data shows that AB-PA01 can reduce biofilms produced by the bacteria Pseudomonas aeruginosa, a major cause of lung infections in cystic fibrosis patients, scientists report. The study,  “Activity of Bacteriophages in Removing Biofilms of Pseudomonas aeruginosa Isolates from Chronic Rhinosinusitis Patients,”…
EBX-001, which consists of two bacteria-fighting compounds, eliminates persistent drug-resistant Pseudomonas aeruginosa in cystic fibrosis patients better than the antibiotic component of the therapy alone, a study reports. The treatment candidate’s developer, EnBiotix, published the results in Antimicrobial Agents & Chemotherapy, a journal of the American Society for Microbiology. The article was…
The benefits of current therapies to treat cystic fibrosis (CF) are limited, so scientists are exploring new strategies based on increasing the expression and activity of alternative chloride channels, such as the ANO1 channel. In a new study, researchers have advanced a potential therapeutic strategy to restore cells’ normal functioning…
Savara Pharmaceuticals has started a Phase 3 clinical trial to evaluate the effectiveness of its AeroVanc inhalant as a treatment for a type of antibiotic-resistant lung infection in cystic fibrosis patients. AeroVanc, also known as vancomycin hydrochloride inhalation powder, is aimed at methicillin-resistant Staphylococcus aureus, or MRSA. There are both gram-positive and gram-negative…
Interim data from an ongoing Phase 2a clinical trial indicate that the investigational therapy MS1819-SD may provide therapeutic benefits for patients with cystic fibrosis (CF) who have exocrine pancreatic insufficiency (EPI), a digestive disorder. The trial (ACTRN12616000962437) is being conducted in patients with chronic pancreatitis (CP) who have EPI, but the drug is being…
The first study of cystic fibrosis (CF) patients who received antibiotics that caused inner ear disease or ototoxicity is enrolling participants, according to Sound Pharmaceuticals. The Phase 1b STOP Ototoxicity study (NCT02819856) is led by Dr. Patrick Flume, MD,…
ProQR Therapeutics says its investigational drug QR-010 improved respiratory symptoms in 70 adults with cystic fibrosis (CF) who participated in a Phase 1b trial of the inhaled therapy. Researchers saw the biggest improvements in patients with a lower lung function at the beginning of the study, which only targeted patients with…
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