The Horizon 2020 research and innovation program of the European Union granted €6.8 million (approximately $7.7 million) to an international research team for the development of non-viral gene delivery technology. This new gene therapy strategy has the potential to improve the health of patients with genetic diseases worldwide, including…
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The European Commission has approved the label extension of Orkambi (lumacaftor/ivacaftor) for the treatment of children ages 2-5 with cystic fibrosis (CF) due to F508del mutations, Vertex Pharmaceuticals, the therapy’s manufacturer, announced. Orkambi was previously approved in Europe to treat CF patients 6 and older with the…
Florida resident Megan Willis has cystic fibrosis (CF), and she’s not getting better. But that didn’t stop authorities last March from deciding otherwise, and denying her access to Medicaid — which for years had been paying her more than $100,000 in annual healthcare expenses. “My health has only gotten worse…
Waiting for a lung transplant in the intensive care unit at a New York hospital in November 2017, a 21-year-old pulmonary hypertension patient named Miriam Holman sued the U.S. Department of Health and Human Services. Her lawsuit accused the department of allocating organs based on a patient’s geographic location,…
A reassessment of U.K. medical records showed that in cystic fibrosis (CF) patients, breathing support provided by gas inhalation, through a face or nasal mask, helps improve lung function but not survival, researchers report. The study “Non-invasive ventilation and clinical outcomes in cystic fibrosis: Findings from…
Sound Pharmaceuticals (SPI) has enrolled its first-patient-in — meaning the first subject meeting the criteria for inclusion and exclusion of a clinical study — in a Phase 2 trial testing SPI-1005 for the prevention and treatment of tobramycin-induced ototoxicity in cystic fibrosis (CF) patients, the company announced. Tobramycin…
A baby born with cystic fibrosis (CF) today can now expect to live a median 44 years, says Preston W. Campbell, MD, president and CEO of the Cystic Fibrosis Foundation (CFF). “Life expectancy for CF patients is going up every year, and I think it’ll reach 70 or 80 in…
Blocking the interaction between two molecules involved in immune restraining mechanisms — called PD-1 and PD-L1 — may be an effective therapeutic strategy to boost the immune system and treat chronic infections of Pseudomonas aeruginosa in cystic fibrosis (CF) patients, according to a recent study. The study, “…
An exploratory Phase 1 trial showed that repeated intranasal treatment with the therapy eluforsen improved CFTR protein activity in adults with cystic fibrosis (CF) carrying F508del mutations in both CFTR gene copies. The study, “Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis,” was…
The World Health Organization (WHO) approved ensifentrine as the recommended name to identify the cystic fibrosis (CF) treatment candidate RPL554. Verona Pharma’s ensifentrine is an inhaled inhibitor of the enzymes phosphodiesterase 3 and 4, designed to have bronchodilator and anti-inflammatory properties. The “-fentrine” staple indicates WHO’s…
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