The Canadian Institutes of Health Research (CIHR) has awarded CA$810,000 (about $637,000) over five years to researchers at the University of Saskatchewan (USask) to support their research exploring the role of two cell types in the lungs of patients with cystic fibrosis (CF). A better understanding…
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The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…
Worldwide, only 12% of people with cystic fibrosis (CF) have access to the triple combination therapy Trikafta, despite this next-generation medicine being suited to a “large proportion” of patients, a study on global rates of CF diagnosis and treatment reported. Global estimates of total CF patients are also likely underestimated due…
Yale University last month launched the Center for Phage Biology and Therapy, a new endeavor to advance research into using viruses to address the growing concern about antibiotic-resistant bacteria. Since the first antibiotics were discovered nearly a century ago, bacteria-killing medicines have revolutionized humanity’s ability to combat bacterial infections. However,…
To continue to support the health of U.S. adults living with cystic fibrosis (CF), the online exercise, education, and well-being platform Beam has extended its collaboration with the Cystic Fibrosis Foundation (CFF) through June. The partnership offers patients free access to live and on-demand training and educational…
It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.
Adults and children with cystic fibrosis (CF) experienced a marked increase in food insecurity — meaning a limited or uncertain availability of foods that are nutritionally adequate and safe — during the COVID-19 pandemic, a U.S. study found. Patients with food insecurity also reported poorer mental health and self-care…
Clustering of the CFTR protein in lipid (fat)-rich platforms on the cell membrane, which can occur when the cell is under stress and increases the number of working CFTR proteins, is disrupted by cystic fibrosis (CF)-causing mutations, a recent study shows. CFTR modulators that are part of Trikafta were…
An international team led by scientists at the University of Toronto identified hundreds of proteins, from among nearly 10,000 screened, that interact with the CFTR protein in mammalian cells. These interacting proteins may be useful therapeutic targets for cystic fibrosis (CF), which is caused by mutations…
The introduction of CFTR modulators — a type of disease-modifying treatment for cystic fibrosis (CF) caused by specific mutations — has brought a general sense of hope to the lives of young adults with the disease, according to a small single-center U.S. study. Notably, patients reported greater physical…
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