News

The European Commission has approved Vertex’s Kalydeco (ivacaftor) for the treatment of children and adolescents with cystic fibrosis (CF) who carry the R117H mutation in the…

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.

In the seven months since Vertex Pharmaceuticals’ triple-combo therapy Trikafta (elexacaftor/tezacaftor/ivacaftor) hit the market, people with cystic fibrosis, their doctors, and the Cystic Fibrosis Foundation (CFF) have all given the drug high marks for effectiveness. Trikafta, a CFTR modulator, is intended to treat CF patients…

Researchers have reported the case of two siblings with impaired CFTR function due to a deficiency in another chloride channel, called TMEM16A, that do not present symptoms associated with cystic fibrosis (CF). While there is evidence supporting either the activation or the suppression of TMEM16A activity as…

Children with cystic fibrosis (CF) have an altered intestinal virome, or viral communities, compared to healthy children that may impact growth and intestinal inflammation, a case-control study reported.  The study, ”The intestinal virome in children with…

Investigational treatment Encala (Lym-X-Sorb) is safe, well tolerated, and increased dietary fat absorption in patients with cystic fibrosis (CF) and pancreatic insufficiency, according to an analysis from a Phase 2 clinical trial. Findings from the analysis were reported in the study, “Improved residual fat malabsorption…

A deputy sheriff’s squad car, lights ablaze and sirens blaring, recently raced to Benjamin Clackum’s house on a sleepy residential street in Lawrenceville, Georgia, but no one was getting arrested. In fact, it was quite the opposite. Clackum was being presented with an AffloVest, a device that would make…

During COVID-19, the U.K. Cystic Fibrosis Registry is taking steps to further the collection of data on how the ongoing pandemic is affecting people with cystic fibrosis (CF). The CF Registry, managed by the Cystic Fibrosis Trust, records data on patients in England, Wales, Scotland, and Northern…

Pravibismane suspension, a new type of inhaled antimicrobial medication developed by Microbion, has received orphan drug designation from the U.S. Food and Drug Administration…

MyCyFAPP, a mobile app designed to help patients with cystic fibrosis (CF) manage their pancreatic enzyme replacement therapy (PERT) regimen, can improve the quality of life of children with the disease, particularly those with severe gastrointestinal (GI) symptoms, study says. The study, “Use…