Alice Melão, MSc,  —

Anthera Pharmaceuticals has enrolled half the patients it is seeking for a Phase 3 clinical trial of Sollpura (lipromatase) as treatment for a cystic fibrosis-related digestive disorder known as exocrine pancreatic insufficiency, or EPI. The company expects to release the key results of the RESULT trial in late 2017 or early…

GeneFo is offering a free online lecture on Sept. 27 about the therapeutic benefits of humor and laughter as strategies to manage disease symptoms in cystic fibrosis. The webinar will provide practical assistance for patients and caregivers on how to enjoy a good laugh, even when living with such a complex disease. Cystic…

Chest X-rays commonly used to monitor cystic fibrosis (CF) patients expose these people to cumulative radiation, a known risk for cancer. A study by researchers in Britain, published in the journal BMJ Open, found that newer equipment lessened this exposure and risk but levels were still a concern, especially for young CF patients…

ProQR Therapeutics has finished a dose-escalating Phase 1b clinical trial evaluating the safety, stability and distribution throughout the body of its investigational therapy QR-010 in cystic fibrosis (CF) patients with a confirmed F508del CFTR gene mutation. Interim top-line results should be released by late September. A missing amino acid in the 508 position of the…

Fluidigm and Baylor Genetics have teamed up to develop a next-generation gene sequencing method that can quickly identify less common mutations of the gene associated with cystic fibrosis. Up to 2,000 mutations can cause CF. The new method will allow doctors to spot those that are less common, improving…

Two of the three therapies that Proteostasis Therapeutics has developed to correct the underlying problem in cystic fibrosis are going through clinical trials and a third is heading for trials, the company reports. That underlying problem is a mutation in a gene known as the cystic fibrosis transmembrane conductance regulator, or CFTR. It produces…

One of the preparations for clinical trials is producing enough of the therapy to be tested. Arch Biopartners says the production of its treatment for antibiotic-resistant bacterial infections in cystic fibrosis and COPD is on track for the start of a Phase 1 clinical trial later this year. Its partner, Dalton…

Results of an online survey, conducted by the cystic fibrosis (CF) community GeneFo, showed that a main concern of CF patients is their intimate lives and sexuality. In an attempt to help CF patients and caregivers to find ways, alone or as a couple, to overcome the physical…

A Phase 3 study of Sollpura (liprotamase), a non-porcine enzyme replacement therapy aiming to treat exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF) better than existing porcine-derived therapies, is now recruiting about 150 pediatric and adult patients in the United States, Europe and Israel. Specifically, the expanded trial will evaluate the efficacy — or non-inferiority — of Sollpura compared…

Forty cystic fibrosis patients or family members are winners of Vertex Pharmaceuticals‘ first All in for CF scholarships. The awards are for $5,000, making the total for the 2017-2018 academic year $200,000. Boston-based Vertex is a biotech company that develops therapies for cystic fibrosis and  other life-threatening conditions. The company…