Marisa Wexler, MS,  senior science writer—

An unusual clinical presentation can hinder proper diagnosis for people with cystic fibrosis (CF), even with newborn screening, a new case report contends. The report, “Uncommon clinical presentation of cystic fibrosis in a patient homozygous for a rare CFTR mutation: a case report,” was published in…

Liver ultrasounds can help identify children with cystic fibrosis (CF) who are at higher risk of developing liver disease, a new study shows. The study, “Heterogeneous Liver on Research Ultrasound Identifies Children with Cystic Fibrosis at High Risk of Advanced Liver Disease: Interim Results of a…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to MRT5005, an investigational RNA-based treatment for cystic fibrosis (CF), the therapy’s developer, Translate Bio, has announced. Fast track is given to medications that aim to treat serious or life-threatening conditions and…

Enrollment is complete for a European initiative to test an organoid-based, personalized-medicine approach to treating cystic fibrosis, with the first trial starting later this year, said Proteostasis Therapeutics (PTI) and CF Europe. The Human Individualized Therapy of CF (HIT-CF) initiative will test PTI drug combinations through…

The genetic programming that causes embryonic cells to differentiate into lung cells was described for the first time in a study in mice. Researchers say that understanding the genetic changes necessary for lung cell development could allow for more effective use of stem cells in research and treatment. The study,…

Enrollment is nearing completion for two Phase 2 clinical trials testing the experimental treatment ELX-02 in people with cystic fibrosis (CF), according to Eloxx Pharmaceuticals, the company developing ELX-02. “We expect full enrollment to be achieved during the first quarter of 2020, and to report topline results…

The Cystic Fibrosis (CF) Foundation awarded a nearly $700,000 contract to Calibr, the drug discovery and development arm of Scripps Research, to support projects aiming to identify compounds that could treat infections caused by Burkholderia cepacia complex. The contract will provide funding for two years, and is valued…

The European Commission has approved a label extension for Kalydeco (ivacaftor), allowing the therapy to be used in infants with cystic fibrosis (CF) who are between the ages of six months and one year. The decision follows a positive opinion on Kalydeco, marketed by Vertex Pharmaceuticals, that…

The Regional Pharmaceutical Procurement Service in Northern Ireland and the National Health Service (NHS) Wales have reached an agreement with Vertex Pharmaceuticals that will allow people with cystic fibrosis (CF) who live there to access all of Vertex’s currently approved medications for the disease. The terms of the…

The Cystic Fibrosis Foundation (CFF) is celebrating the United States Food and Drug Administration’s decision to approve Trikafta. The therapy — a triple combo based on elexacaftor, tezacaftor, and ivacaftor, which is being developed by Vertex Pharmaceuticals — was approved on Oct. 21 for people with cystic fibrosis (CF)…