News

The U.S. Food and Drug Administration (FDA) has given fast track designation to ELX-02, an investigational treatment for people with cystic fibrosis (CF) caused by nonsense mutations. This FDA designation aims to speed the development and regulatory review of potential treatments for serious or life-threatening diseases with an unmet medical…

Differences in Psl, a complex sugar molecule produced by Pseudomonas aeruginosa, contribute to the bacterium’s resistance against the antibiotic tobramycin in children with cystic fibrosis (CF), a study shows. While tobramycin-resistant P. aeruginosa produced similar levels of Psl to those that were effectively eradicated with the antibiotic, its Psl molecules…

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

The Centers for Medicare and Medicaid Services (CMS) has published a local coverage determination confirming that Relizorb — an enzyme cartridge to help in digestion for cystic fibrosis (CF) patients receiving their nutrition through enteral feeding — will be covered by Medicare.

Blacks and Hispanics with cystic fibrosis (CF) have worse lung function than white patients, according to a study from New York City. However, no differences were found among these groups of patients in several other parameters, including the number of visits to the doctor, treatments with antibiotics, and hospitalizations. The…

EnBiotix has purchased murepavadin, an investigational inhaled antibiotic to treat Pseudomonas aeruginosa infections in people with cystic fibrosis (CF), from Polyphor. The two companies also entered into an agreement to merge, wherein Polyphor will acquire all EnBiotix stock in exchange for Polyphor stock newly issued upon closing. The merger…

The federal agency that administers Medicare programs in the U.S. has just made it easier for people with cystic fibrosis (CF) to get the nutritional supplement Encala, designed to help CF patients better absorb fat in their digestion. The Centers for Medicare and Medicaid Services, called CMS, has…

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…

Cystic fibrosis (CF) patients, caregivers and supporters are invited to participate in Rose Up, a Sept. 17 virtual event aimed at uniting the CF community and raising awareness and funds to help find a cure. The hope is that the Cystic Fibrosis Foundation (CFF) effort, now in its…

An assessment called the lung clearance index could be used to help identify people with cystic fibrosis (CF) who are at high risk for worsening lung function, a study has found. The study reported on factors associated with a worsening lung clearance index (LCI) over time in CF patients,…