News

Cystic Fibrosis Canada has opened an initiative called Elevate, inviting people affected by cystic fibrosis (CF) to share their experiences, perspectives, and priorities with the nonprofit group.  “We have a history of asking for input from the community, but we felt…

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

Pharmacy benefits and services company Elixir has chosen Maxor Specialty Pharmacy to be its network’s exclusive provider of medications for cystic fibrosis (CF). Maxor Specialty specializes in rare diseases like CF and is one of four pharmacies in the U.S. to have access to all medications approved for cystic fibrosis,…

A research ethics committee in Australia has given Krystal Biotech the go-ahead to start a Phase 1 clinical study of KB407, the company’s investigational inhaled gene therapy for cystic fibrosis (CF). Enrollment for the trial is expected to start soon, according to Krystal, following approval from the…

The publicly funded drug access programs in the Canadian provinces of Alberta and Saskatchewan are now covering the triple-combination therapy Trikafta for eligible patients with cystic fibrosis (CF). These provinces join Ontario as the first to confirm coverage of Trikafta in Canada. “It’s a relief to know that…

The implementation of newborn screening for cystic fibrosis (CF) in the U.S. allowed for early diagnosis and clinical evaluation of affected infants, and reduced rates of serious lung infections and hospitalizations over time, according to data from the first nine years of universal screening. However, early nutritional deficits…

People with cystic fibrosis (CF) in Ontario, Canada, will now have access to the triple-combination therapy Trikafta through the province’s publicly funded drug program. “Our government has taken urgent action to ensure all cystic fibrosis patients will have more timely access to the effective and lifechanging treatments they need,”…

Health Canada has granted priority review to Vertex Pharmaceuticals’ application seeking the approval of Kalydeco (ivacaftor) to treat cystic fibrosis (CF) patients, ages 4 months to 18 years, carrying the R117H mutation in the CFTR gene. The intended label extension is for patients who weigh at least 5 kilograms…

A non-invasive ultrasound imaging technique may be able to detect changes in the pancreas of children with cystic fibrosis (CF) and aid in an early diagnosis of exocrine pancreatic insufficiency, or EPI — which occurs when the pancreas doesn’t make enough digestive enzymes — a Turkish study suggests.

Scientists using phage therapy — specifically a bacteriophage they dubbed “Muddy” — successfully treated an infection by an antibiotic-resistant strain of Mycobacterium abscessus bacteria in a zebrafish model of cystic fibrosis (CF). For five days, the team treated the zebrafish, which were infected with M. abscessus, a lung-damaging bacteria…