Using the CRISPR gene editing tool, a team of researchers was able to permanently correct two mutations in the CFTR gene — mutations that cause cystic fibrosis (CF) — in CF patient-derived cells. The researchers showed that the corrected CFTR gene was functional and working normally in mini-intestines called…
cystic fibrosis
A 10th anniversary screening of the documentary “65_RedRoses” will take place on Sunday, Sept. 8, at the Vancouver Playhouse Theatre to raise funds for the Transplant Research Foundation of British Columbia (TRFBC). “65_RedRoses” is an award-winning documentary that was directed by Nimisha Mukerji and Philip Lyall. It follows Eva…
CF Foundation Earmarks Up to $15M for Arcturus Therapeutics to Advance Experimental RNA Therapy
The Cystic Fibrosis Foundation (CFF) will give up to $15 million to Arcturus Therapeutics to advance the development of an experimental RNA therapy, called LUNAR-CF, to treat CF patients with all types of mutations in the CFTR gene. The funding will establish a multi-year program aimed at boosting the development…
Italfarmaco SpA entered an agreement with Neupharma to develop and commercialize an inhaled formulation of teicoplanin, one of the most effective antibiotics to treat lung infections caused by resistant strains of Staphylococcus aureus in people with cystic fibrosis (CF). Under the terms of the agreement, Italfarmaco shall hold…
Eloxx Pharmaceuticals is preparing to launch a Phase 2 clinical study in the U.S. to explore the potential of ELX-02 for treating people with cystic fibrosis (CF) who carry the G542X mutation. This follows approval by the U.S. Food and Drug Administration (FDA) of the…
A single dose of MRT5005, Translate Bio‘s first-in-class messenger RNA (mRNA) therapy for the treatment of cystic fibrosis (CF), improves patients’ lung function, interim data from a Phase 1/2 trial shows. While stressing these are early results, the researchers called the findings “promising.” CF is a…
Treatment for three years with Kalydeco (ivacaftor) reduces the risk of lung infection by serious pathogens — including Pseudomonas aeruginosa and Staphylococcus aureus — in people with cystic fibrosis (CF), a U.K. study reports. The study, “Ivacaftor Is Associated with Reduced Lung Infection by Key Cystic Fibrosis…
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health coverage. As if…
People using a nebulizer while lying on their side showed no significant loss in a treatment’s overall deposition in the lungs, a study in healthy adults and in cystic fibrosis (CF) patients reports. Rather, it found that alternating sides instead of sitting upright may better deposit a medicine’s active compounds…
Employees at the Development Bank of Wales, in the U.K., have raised £30,400 (around $37,000) to support the Cystic Fibrosis Trust. Every year, bank employees are asked to nominate a charitable organization to support, with which they have a personal connection. This year, the Cystic Fibrosis Trust was…
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