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Topline data from a Phase 2b trial showed that the investigative therapy acebilustat taken once daily can reduce the number and frequency of pulmonary exacerbations in patients with cystic fibrosis (CF). It did not, however, seem to affect patients’ overall lung function. Pulmonary exacerbations — temporary worsening of lung function — are…

A previously undiscovered and rare type of cell has been identified in the tissue lining the airway of the lungs and carrying high levels of the CFTR gene, the mutation of which is the underlying cause of cystic fibrosis (CF), a study reports. While the exact role of…

Cystic fibrosis is (CF) patients receiving higher doses of pancreatic enzyme replacement therapy (PERT) gain more weight, a recent report shows. PERT is a tablet that contains pancreatic enzymes. When taken with food, this therapy can improve the absorption of necessary nutrients. Many CF patients struggle with weight gain. This is…

The Q359K/T360K gene mutation, which is commonly found in Jewish patients of Georgian descent, is associated with a particularly severe cystic fibrosis (CF) phenotype, according to researchers. Their study, “The Q359K/T360K mutation causes cystic fibrosis in Georgian Jews,” was published in the Journal of Cystic Fibrosis.

A high number of sudden disease worsening episodes — pulmonary exacerbations — and associated increases in healthcare costs were observed in Medicaid patients with cystic fibrosis (CF), according to researchers. Their study, “The burden of cystic fibrosis in the Medicaid population,” was published in the journal ClinicoEconomics…

Vertex Pharmaceuticals’ Symkevi (tezacaftor/ivacaftor) is one step closer to being approved in the European Union as a treatment for cystic fibrosis (CF) patients ages 12 or older who have certain CFTR genetic mutations. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency…

Vertex Pharmaceuticals plans to conclude the enrollment of patients in its Phase 3 programs involving its triple combo therapies to treat cystic fibrosis (CF) in the second half of 2018, the company recently announced in a corporate update. This is earlier than expected for the four Phase 3 trials, which…

Babies with cystic fibrosis (CF) are more likely to be born preterm — less than 37 weeks of pregnancy — and have significantly lower birth weight, according to a large study from Denmark and Wales. The study “Impact of cystic fibrosis on birthweight: a population based study of children…

Arcturus Therapeutics’ RNA-based approach enables the production of healthy CFTR — the protein whose dysfunction is key in cystic fibrosis (CF) — and can be effectively delivered into lung cells, according to the company’s preclinical results. This CF treatment candidate is being developed in collaboration with Cystic Fibrosis…

Grace Rose, a 15-year-old designer who was born with cystic fibrosis (CF), will host the 15th Annual Grace Rose for Cystic Fibrosis Fashion Show Fundraiser on Aug. 18 in Los Angeles to raise funds and awareness about the disease, according to a press release. Taking place at the…