Marisa Wexler, MS,  senior science writer—

A potential gene therapy for cystic fibrosis, KB407, is able to induce the expression of normal CFTR protein in cell models of CF, its developer, Krystal Biotech, reports. These data are in the poster, “In vitro Pharmacology of KB407, an HSV-1 based gene therapy vector,…

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion for a label extension of the cystic fibrosis (CF) treatment Kalydeco (ivacaftor). If approved by the European Commission, the extension would allow Kalydeco use in patients 6…

Vertex Pharmaceuticals announced a multiyear partnership with Affinia Therapeutics to develop gene therapies for cystic fibrosis (CF) and other genetic disorders. Gene therapy seeks to replace a malfunctioning gene with a working version. In people with CF, in particular, a gene therapy would deliver a healthy copy of the…

Orkambi (ivacaftor/lumacaftor) can improve glucose tolerance in people with cystic fibrosis, a small study in patients either at risk of or newly diagnosed with diabetes suggests. The study, “Effect of one-year lumacaftor–ivacaftor treatment on glucose tolerance abnormalities in cystic fibrosis patients,” was published in the …

Supplemental estrogen, most commonly taken for contraception, is associated with poorer bone mineral density in people with cystic fibrosis (CF), a new study suggests. The study, “Oral ethinyl estradiol treatment in women with cystic fibrosis is associated with lower bone mineral density,” was published in the …

A measure called the lung clearance index (LCI) can predict low blood oxygen levels during sleep in people with cystic fibrosis (CF) better than conventional assessments of lung function, a study suggests. The study, “Lung clearance index evaluation in detecting nocturnal hypoxemia in cystic fibrosis patients:…

Delivering nutrients through dialysis could help people with co-occurring cystic fibrosis (CF) and kidney disease meet their nutritional requirements, a recent case report illustrates. The report, “Intradialytic parenteral nutrition improves nutritional status in a complex cystic fibrosis patient with redo double lung transplant and end-stage renal…

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…