Long-term use of Orkambi (lumacaftor/ivacaftor), an approved cystic fibrosis (CF) treatment, is safe and effective for children between ages 2 and 5 with two copies of the disease-causing F508del mutation in the CFTR gene, a study reported. It draws on results from a Phase 3 open-label extension trial…
cystic fibrosis
To mark its 10th anniversary, Claire’s Place Foundation has launched a website that seeks to connect the community of people affected by cystic fibrosis (CF). “Our new website is a supportive forum for inspiration as well as a resource to learn about developments in CF, our programs and…
An ex vivo technique using nasal epithelial cells from a young girl carrying a rare mutation profile for cystic fibrosis (CF) was successful at predicting the efficacy of an approved CF therapy, a case report shows. Researchers first tested the effectiveness of Orkambi (lumacaftor/ivacaftor) on the child’s own…
A clinical trial underway in Israel is investigating how well a personalized chest physiotherapy system compares with traditional airway clearance techniques in people with cystic fibrosis (CF) and other chronic lung conditions, a company reports. “This trial brings us one step closer to our goal: improving the lives of millions…
Olgram has raised €1.5 million (about $1.77 million) in seed funding from investors to speed its development of antibiotics to clear persistent bacteria responsible for chronic, repeat infections. The company’s immediate focus is cystic fibrosis (CF), a disease characterized by chronic bacterial infection in the lungs. In a…
Thirona announced it has developed a new software program that can quickly detect and quantify lung anomalies in cystic fibrosis (CF) patients, including those beyond the capacity of a doctor in a clinic. The software is based on an artificial intelligence (AI) algorithm, called PRAGMA-AI, that is reported…
Health regulators in Australia have approved Trikafta (elexacaftor/tezacaftor/ivacaftor) to treat people with cystic fibrosis (CF), ages 12 years and older, who have at least one copy of the F508del mutation in the disease-causing gene, Vertex Pharmaceuticals, the therapy’s developer, announced. “We are delighted” that Trikafta is now…
Trikafta, a medication that combines three different therapies (elexacaftor/tezacaftor/ivacaftor), is safe and effective in children with cystic fibrosis (CF) ages 6 to 11 who have certain genetic mutations, results from a Phase 3 clinical trial show. The U.S. Food and Drug Administration (FDA) recently accepted for review a…
Parents of children and young adults with cystic fibrosis (CF) in Ontario sent a video appeal to the head of their province’s government, asking the premier to quickly expand prescription criteria that limits their access to key therapies by Vertex Pharmaceuticals. Two of the three gene modulating treatments noted…
Translate Bio’s ongoing Phase 1/2 clinical trial investigating MRT5005, its candidate inhalation therapy for cystic fibrosis (CF), found that repeat dosing is generally safe and well-tolerated. MRT5005 is designed to treat CF by delivering messenger RNA (mRNA) with instructions for cells to produce a working CF transmembrane conductance regulator…
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