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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…

Preventing a protein called AGTR2 from going about its work — either by removing it through genetic engineering or by using pharmacological agents to silence it — improved lung function in mouse models of cystic fibrosis (CF), a study reports. Earlier work linking this protein to pulmonary disease in CF patients,…

Inhaled nitric oxide (NO) was shown to be an effective antibacterial agent against Mycobacterium abscessus infection in preclinical studies, as well as in a pilot clinical trial, according to AIT Therapeutics. The company discussed the latest data on its NO product in two poster presentations during the 3rd Annual…

Five scientists whose work has been instrumental in developing disease-modifying treatments for cystic fibrosis (CF) are recipients of the 2018 Warren Alpert Foundation Prize. Until recently, therapies for CF targeted only the symptoms of the disease. The U.S. Food and Drug Administration (FDA) approved the first disease-modifying therapy, Kalydeco (ivacaftor, marketed…