Author Archives: Marisa Wexler MS

A two-day virtual event called BreatheCon will offer adults with cystic fibrosis (CF) an opportunity to connect and share their experiences through open and honest dialogue. Registration for the Sept. 24–25 event, which is hosted by the Cystic Fibrosis Foundation, is free online. “CF can be extremely…

Increasing the levels of the SFPQ protein helped to restore expression and function of the CFTR protein in cells with a F508del mutation in the CFTR gene, the most common cause of cystic fibrosis (CF). Work also showed that SFPQ levels were unusually low in these cells, suggesting this protein “may play…

A new study is seeking to understand whether and how the symptoms of cystic fibrosis (CF) are affected by the menstrual cycle. To that end, CF patients ages 18 to 45, who have regular monthly periods, are being sought to participate in the aptly named MENSTRUAL study —…

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…

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.

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…

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,…

The triple-combination therapy Trikafta significantly improved lung function and life quality in a global Phase 3 trial that enrolled people with cystic fibrosis (CF) caused by one F508del mutation, and one gating mutation or one residual function mutation, in the CFTR gene. Data from the…

Health Canada has extended the use of Kalydeco (ivacaftor) to treat babies, starting at 4 months old, who have cystic fibrosis (CF) caused by certain mutations. The specific gating mutations covered are: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. Kalydeco, Vertex Pharmaceuticals, was previously approved in…