Magdalena Kegel,  —

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…

4D Molecular Therapeutics (4DMT) announced it has secured funding to complete studies necessary for its gene therapy candidate for cystic fibrosis (CF), 4D-710, to be tested in people. The therapy is composed of a what 4DMT describes as an advanced and customized adeno-associated virus (AAV) vector, which will carry…

Good nutrition may slow the progression of cystic fibrosis patients’ lung disease, according to a review by researchers at the University of Oklahoma Health Sciences Center. It is especially important that cystic fibrosis patients with an end-stage lung disease not limit their consumption of healthy food, even if they…

Some children with severe infections who require intravenous antibiotics can be treated safely at home, according to a review of previously published studies. The review in the journal The Lancet Infectious Diseases reported that none of the studies found that home-based treatment was riskier than hospital care. The review also…

Scientists have long known that defects in the CFTR protein cause cystic fibrosis. Now they’ve discovered that the defective protein leads to faulty insulin signaling, which, in turn, can cause diabetes and also contribute to cystic fibrosis complications. Insulin is a hormone that regulates metabolism, or the conversion of food to…

Vertex Pharmaceuticals is continuing to develop treatments for all people with cystic fibrosis (CF), with clinical trials exploring combinations of its potentiator and corrector therapies. At the same time, efforts to expand the use of already-approved treatments are ongoing. In addition, the company recently completed the purchase of Concert’s…

Relizorb, an enzyme-containing cartridge that breaks down fats in tube-feeding formulas, is now available to children, according to its developer, Alcresta Therapeutics. The U.S. Food and Drug Administration, which approved the device’s use in adults in 2015, has widened the approval to children as young as 5 years old, Alcresta said The…

The U.S. Food and Drug Administration (FDA) has approved Digestive Care’s Pertzye (pancrelipase) to be given to cystic fibrosis patients through a so-called G-tube — a feeding tube delivering nutrition directly to the stomach through a hole in the abdomen. Pertzye is an enzyme replacement therapy for exocrine pancreatic…

Three triple-combination treatments from Vertex Pharmaceuticals showed promising results in Phase 1 and Phase 2 clinical trials in cystic fibrosis (CF) patients with one F508del mutation and one so-called minimal function mutation (F508del/Min) in the CFTR gene, the defective gene that causes CF. Vertex announced that these are the first data to demonstrate…

The U.S. Food and Drug Administration is promoting a targeted treatment approach known as precision medicine to help people with diseases stemming from specific, and often rare, genetic features. Its efforts include expanding the use of approved therapies to other genetic-based conditions, and pushing for the development of more biomarkers…