cystic fibrosis

Organoid technology has created hopes of improved, personalized treatment for cystic fibrosis, but has also posed challenges and generated doubts. Organoids are three-dimensional, engineered cell structures built from stem cells that mimic the architecture and function of human tissue. These mini-organs have the potential to improve our knowledge of disease-related…

A lot of women with a chronic disease like cystic fibrosis fail to receive enough information from doctors on preventing or facilitating pregnancy, an Australian study reports. The result is that many of the four million women in this category become pregnant young, have an unintended pregnancy, or an abortion,…

Long-term prognosis of cystic fibrosis (CF) can be anticipated by analyzing a set of 10 genes related to immune response, researchers at National Jewish Health say. These findings were reported in the journal Annals of the American Thoracic Society, in an article titled “Whole Blood Gene Expression Profiling…

Researchers have linked variations in the mix of microorganisms in cystic fibrosis patients’ airways to their disease outcomes. The findings in the journal PLOS One were in an article titled “Fluctuations in airway bacterial communities associated with clinical states and disease stages in cystic fibrosis.” CF patients typically have particular…

The Cystic Fibrosis Foundation is giving Laurent Pharmaceuticals $2 million to conduct a Phase 2 clinical trial of Laurent’s cystic fibrosis therapy LAU-7b. The additional funding comes on top of $3 million that the foundation has already given the company. “We are truly grateful for this additional support from Cystic Fibrosis…

Genetic sequencing and the speed with which it can help diagnose a child’s disease — in addition to revealing the genes that cause at least half of the 7,000 rare diseases currently known — was the focus of a discussion by three top New York geneticists. The Feb. 28 conference,…

Key results of a Phase 1/1b clinical trial of Pulmatrix’s lead treatment for fungal infections in asthma and cystic fibrosis (CF) are expected by mid-2018, the company reports. The trial, which started in February, is focusing on pulmazole’s safety and patients’ ability to tolerate it. Pulmatrix expects the results to…

The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to Proteostasis Therapeutics’ cystic fibrosis therapy PTI-428 — a move aimed at accelerating its development. Mutations of the CFTR gene cause CF. The most common is F508del, which leads to faulty CFTR protein. Normal CFTR protein is required for transporting chloride ions…

When it comes to rare diseases, one that definitely makes the list is spinal muscular atrophy with respiratory distress — SMARD,  for short. Hunter Pageau, a 12-year-old boy from North Haven, Connecticut, is one of only 80 people in the world known to have SMARD, a motor neuron disease…

Dozens of diverse types of the bacteria Staphylococcus aureus were identified in the airways of cystic fibrosis (CF) patients, including five new types and one commonly associated with livestock, a study reports. Findings were reported in the study, “Emergence and spread of worldwide Staphylococcus aureus clones among cystic fibrosis patients, published in the…