News

The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) for the treatment of infants from six to 12 months old, who have cystic fibrosis (CF) and at least one mutation in the CFTR gene, announced the therapy’s developer, Vertex Pharmaceuticals. With the potential to treat the underlying…

Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how much harder it is for patients with rare diseases who must get to doctors’ appointments or clinical trials that are hundreds of miles away from home.

Normalizing the acidity in the thin layer of liquid covering the airways of newborns with cystic fibrosis might make them more resistant to bacterial infections, a study has found. The study’s researchers also identified two proteins, pendrin and ATP12A, that could be targeted to regulate acidity levels of the airway liquid in these…

Physical and signaling interactions between two pathogen groups that often affect patients with cystic fibrosis (CF) — Pseudomonas aeruginosa bacteria and Scedosporium fungi — result in a growth effect (inhibition or enhancement) of these microorganisms, according to an in vitro study. CF therapies such as antibiotics or corticosteroids also affected the growth of the fungi, the…

Having to take insulin to treat cystic fibrosis-related diabetes (CFRD) adds to patient distress and sense of a poorer quality of life, a study says, alerting experts to a need to find approaches that might ease this burden. The findings were…

The not-for-profit organization TB Alliance will receive up to $5.1 million from the Cystic Fibrosis Foundation (CFF) to advance the discovery and development of treatments for multidrug-resistant nontuberculous mycobacteria (NTM) infections in people with cystic fibrosis (CF). TB Alliance will partner with Johns Hopkins University over the next three years…

The Cystic Fibrosis Foundation (CFF) recognized CARSTAR — the largest franchise network of auto body repair service in North America — as one of its National Corporate Champions of the year for raising more than $100,000 toward cystic fibrosis (CF) research and care. CARSTAR also received the Rising Star…

With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…

Using the gene editing tool CRISPR-Cas9 to specifically target the lung of a developing fetus, researchers were able to correct a mutation associated with a human lung disease that is fatal after birth, a study in mice shows. These findings highlight the potential of gene editing strategies to correct mutations…

The early-stage venture capital fund AmorChem has entered an agreement with Université de Sherbrooke (UdeS) and TransferTech Sherbrooke, in Canada, to optimize the antibiotic tomatidine for treating infections associated with cystic fibrosis (CF). Tomatidine is a naturally occurring product that can be extracted from tomatoes. Previous findings have…