cystic fibrosis

Rapid weight gain early in life may have a long-term negative impact on the growth of children with cystic fibrosis (CF), a study has found. This finding suggests that nutritional interventions aimed at supporting the child’s normal growth should be implemented early on, taking into consideration their weight…

The case report of a cystic fibrosis (CF) patient suggests that phages — viruses that naturally infect and kill bacteria — are a potential personalized therapy against infections caused by antibiotic-resistant bacteria. The study, “Engineered bacteriophages for treatment of a patient with a disseminated drug-resistant Mycobacterium…

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…

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…

Motif Bio has entered an agreement with Lamellar Biomedical to undertake a preclinical study that will test a new therapeutic combination for treating lung infections in patients with cystic fibrosis (CF). Under the terms of the agreement, Motif Bio will launch a preclinical study to assess the effectiveness…

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 non-profit organization Emily’s Entourage is teaming up with event coordinator Eesho for its 4th Annual Emily’s Entourage New York, a fundraiser for new treatments and a cure for cystic fibrosis (CF). Known as “EENY: Ultra Violet,” the talent-laden event will begin at 7 p.m.  Saturday, May 4,…

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…

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…