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…
Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…
Vertex Pharmaceuticals has signed a letter of intent with the pan-Canadian Pharmaceutical Alliance (pCPA), which is an agreement in principle to extend public reimbursement to Trikafta, a triple-combination therapy for eligible cystic fibrosis (CF) patients. The extension adds to the letter of intent (LOI) with the pCPA for…
AirPhysio, a handheld device designed to help clear the mucus that builds up in the lungs of people with cystic fibrosis (CF), is now available online in the U.S. through Life Wellness Healthcare. The Australian-based company said in a press release that the AirPhysio — which…
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…
A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…
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