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,…
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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…
An inhaled form of messenger RNA (mRNA) with the potential to safely deliver therapeutics to the lungs of cystic fibrosis (CF) patients has been discovered by researchers at the Massachusetts Institute of Technology (MIT). Researchers developed a type of nanoparticle given as an inhaled mist (nebulizer) that allows the…
Vertex Pharmaceuticals and Arbor Biotechnologies have established a partnership to discover new proteins to advance the development of gene therapies for cystic fibrosis. The companies also plan to develop new genome-editing approaches for four other diseases but haven’t revealed which ones yet. Under the terms of the…
The real estate investment and management firm MC Companies, whose owners are grandparents of children with cystic fibrosis, was recognized for its support of disease research by The Cystic Fibrosis Foundation – Tucson Chapter at its fourth annual Round-Up for a Cure event. Round-Up for a Cure is a yearly fundraising event, put…
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