News

The Australian Medical Services Advisory Committee (MSAC) has recommended that the costs of screening for mutations known to cause  cystic fibrosis (CF), spinal muscular atrophy (SMA), and fragile X syndrome be open to reimbursement for couples planning or in the early stages of pregnancy. Its favorable opinion…

Machine learning, a method of data analysis, could improve disease prediction and bring precision medicine for cystic fibrosis (CF) one step closer to reality, according to researchers at the University of Cambridge, in the U.K., who have developed novel artificial intelligence (AI) technologies to allow for clinical…

Oral lenabasum failed to significantly reduce the frequency of pulmonary exacerbations in people with cystic fibrosis (CF) treated in a Phase 2 trial, but new analyses suggest this failure could be attributed, at least in part, to differences among the patients that confounded results. The investigational therapy showed some…

Eloxx Pharmaceuticals presented preclinical safety and efficacy data supporting ELX-02, its investigational treatment for cystic fibrosis (CF) caused by nonsense mutations that is also being evaluated in two enrolling, Phase 2 trials. The data were presented as two posters at this year’s virtual North American Cystic Fibrosis…

The National Board for Respiratory Care (NBRC) has launched “CHOOSE ONE: Every Breath Counts Campaign,” a fundraising initiative intended to protect and improve the lives of people who need respiratory care, such as those with lung diseases like cystic fibrosis (CF). This year, more than $200,000 will…

The Cystic Fibrosis Foundation (CFF) is awarding Synspira Therapeutics up to $14 million in additional funding to support the development of two therapies to address complications of cystic fibrosis (CF), namely malabsorption and pulmonary infections. “Despite the approval of new treatments for the underlying cause of CF, many…

Inhaled tobramycin alone works as well at eradicating an initial infection by Pseudomonas aeruginosa bacteria in people with cystic fibrosis (CF), and its use in combination with an oral fluoroquinolone, such as ciprofloxacin or levofloxacin, appears to be redundant, a small study reported. Its results also suggest that inhaled…

The U.S. Food and Drug Administration (FDA) cleared Armata Pharmaceuticals‘ investigational new drug (IND) application for a Phase 1b/2a clinical trial of AP-PA02 for the treatment of the Pseudomonas aeruginosa bacterial infections that are a hallmark of cystic fibrosis (CF). “We are very pleased…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

A Phase 2 clinical trial investigating MS1819 in combination with stable pancreatic enzyme replacement therapy (PERT) to treat exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF) is actively recruiting participants in Europe, AzurRx BioPharma, the treatment’s developer, announced. The open-label study (NCT04302662) expects to enroll 24 CF patients, ages 12…