Women with conditions such as cystic fibrosis (CF) who planned their pregnancies after a lung transplant experienced better outcomes compared with those who did not plan their pregnancies, according to a recent study. Those positive outcomes included longer gestation periods, higher birth weights of their babies, and a lower…
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Delivering gene-editing tools to mice while they were still developing in the womb corrected the mutated CFTR gene that causes cystic fibrosis (CF), with the correction lasting into adulthood and restoring normal protein function in the lungs and digestive system, a study found. While it’s still too early…
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…
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…
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