Alyftrek has been approved by the European Commission to treat cystic fibrosis (CF) patients 6 years and older who have at least one non-class 1 mutation in the CFTR gene. This indication in the European Union (EU) represents the broadest label for Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) in the world, according to the…
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More than a third of caregivers of children with cystic fibrosis (CF) reported delays in getting information about the diagnosis after a positive newborn screening (NBS) test for the disease, according to a study in the U.S. Caregivers mentioned delays in receiving their child’s CF diagnosis and in…
Adding the experimental treatment CMTX-101 to standard antibiotics reduced the number of Pseudomonas aeruginosa — a type of bacteria often involved in serious infections — in the lungs of people with cystic fibrosis (CF), while also being safe and well tolerated. These are interim findings from 21 patients…
A lack of stability in a specific region of the CFTR protein may explain why certain people with cystic fibrosis (CF) don’t respond to available modulator therapies, a new study suggests. The findings imply drugs to stabilize this specific region — some of which are already in clinical development…
Two experimental CFTR modulator therapies being developed by Sionna Therapeutics are able to improve the function of the most common mutated form of CFTR protein that causes cystic fibrosis (CF). That’s according to new data Sionna presented at the European Cystic Fibrosis Society (ECFS) 48th European Cystic Fibrosis…
Although infants with cystic fibrosis (CF) may have lung function abnormalities between the ages of 3 months and 2 years, these are predominantly mild and temporary, a real-world study reports. Tests that indicate abnormal lung function may influence decisions about clinical management of CF. Clinical findings, however, including evidence…
SION-719 and SION-451, two NBD1 stabilizers Sionna Therapeutics is developing to restore CFTR protein function in cystic fibrosis (CF), will advance to the next stages of clinical development after positive safety results in Phase 1 clinical trials. Sionna tested both compounds in healthy volunteers to assess safety and…
While most adults with cystic fibrosis (CF) view their health as very good or excellent while on Kalydeco (ivacaftor) or Trikafta (elexacaftor/tezacaftor/ivacaftor) — both approved CFTR modulator therapies for CF — about 1 in 7 still report their health as fair or poor, a survey study…
Using inhaled antibiotics can help control the growth of certain bacteria in the lungs, but it may increase the risk of fungal infections with Aspergillus fumigatus in people with cystic fibrosis (CF), according to a study. The study, “Association between inhaled antibiotic use and treatment-emergent organisms among Canadian people with cystic fibrosis,”…
The Cystic Fibrosis Foundation (CFF) has awarded $5 million to the Cleveland Clinic to continue as the biorepository coordinating center for the Cystic Fibrosis Lung Transplant Consortium until 2030, supporting research and other efforts to improve lung transplant outcomes. Created in 2017, the consortium has established a…
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