News

The RESTORE-CF clinical trial investigating Translate Bio’s candidate therapy MRT5005 has completed the single-ascending dose regimen in patients with cystic fibrosis (CF), the company announced. The ongoing Phase 1/2 trial (NCT03375047) is testing the safety and efficacy of single and multiple escalating doses of nebulized MRT5005, compared with each other and…

Abeona Therapeutics‘ new gene therapy ABO-401 for the treatment of cystic fibrosis (CF) has successfully delivered a functional version of the CFTR gene to the lungs of mice with CF, according to a preclinical study. In addition, the therapy showed the potential to restore the function of CFTR protein…

Specific Pseudomonas aeruginosa bacteria called mucoid variants, which are linked to poor prognosis of patients with cystic fibrosis (CF), are associated with significantly greater regional lung inflammation, a study has found. The study, “Mucoid Pseudomonas aeruginosa and Regional Inflammation in the Cystic Fibrosis Lung,” was published in the…

Former National Football League quarterback Boomer Esiason, whose son has cystic fibrosis (CF), will be a keynote speaker at the National Association of Specialty Pharmacy’s (NASP) Annual Meeting & Expo. One of the most high-profile figures in the fight against CF, Esiason will speak on the opening…

A pharmaceutical sciences researcher will use a five-year, $3.3 million grant from the National Institutes of Health (NIH) to develop an inhaled therapy for use by all cystic fibrosis (CF) patients, regardless of the genetic mutation underlying their disease. Researcher Gaurav Sahay, PhD, of Oregon State University (OSU),…

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…