cystic fibrosis

Patient enrollment and dosing has resumed in a Phase 1/2 clinical trial testing MRT5005, Translate Bio‘s potential messenger RNA (mRNA) therapy for people with cystic fibrosis (CF) regardless of their underlying mutation. The trial, called RESTORE-CF trial (NCT03375047) and enrolling up to 40 adults with stable disease at 17 centers…

The NuvoAir Home platform — NuvoAir’s respiratory support system for people with lung diseases — allows adults and children with cystic fibrosis (CF) to gather high quality data on the health of their lungs at home and without medical supervision. Adherence to NuvoAir’s respiratory system, and its…

The Cystic Fibrosis Foundation (CFF) has awarded up to $2.4 million to Calithera Biosciences to advance in testing CB-280, its investigative oral therapy that aiming to lower the risk of lung infections in people with cystic fibrosis (CF). An enrolling clinical trial is underway in…

The European Commission has approved Kalydeco (ivacaftor), by Vertex Pharmaceuticals, to treat infants as young as four months old with cystic fibrosis (CF) caused by certain mutations. The decision to extend Kalydeco’s label to younger babies, weighing at least 5 kg (about 11 lbs) and with relevant mutations, follows…

The Cystic Fibrosis Foundation (CFF) has awarded funding to enGene to support its work on a non-viral and inhalable gene therapy that could deliver a working copy of the CFTR gene to the lungs of people with cystic fibrosis (CF). “Today’s announcement reflects yet another milestone on our path…

Pseudomonas aeruginosa mutate into a mucoid form in the lungs of people with cystic fibrosis (CF), creating biofilms that render these bacteria more resistant and more damaging, a study reported. Its researchers suggest a way of studying P. aeruginosa cells to identify treatments…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

Envara Health has launched Encala (formerly Lym-X-Sorb), a nutritional supplement designed to improve fat and nutrient absorption in people with cystic fibrosis (CF) and pancreatic insufficiency. Encala is indicated for adults and children older than 1 who have started eating toddler or family table food. According…

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…