Marisa Wexler, MS,  senior science writer—

The Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor) as a treatment for cystic fibrosis (CF) in people 12 and older who have either two F508del mutations or one F508del mutation and…

Nontuberculous mycobacteria (NTM), a type of infectious bacteria, is able to survive being dried for a full day, a study found, suggesting that drying is not a sufficient way to disinfect nebulizers used by people with cystic fibrosis (CF). The study, “Susceptibility of the Mycobacterium…

Most people in the U.K. are unaware of the challenges faced by people living with cystic fibrosis (CF), according to results of a YouGov poll commissioned by the U.K. Cystic Fibrosis Trust. Data from the survey indicates that four of five people in the general public do not…

The third annual CF FamilyCon, a free and online event that connects people with cystic fibrosis (CF) and their extended families and friends with one another — and to others — takes place this weekend. This two-day event, supported by the Cystic Fibrosis Foundation, is set for…

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…

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 …