Savara Pharmaceuticals has stopped patient enrollment into two of its ongoing clinical trials in cystic fibrosis (CF) patients — AVAIL and ENCORE — for safety reasons due to the COVID-19 pandemic. Steps will also be taken to ensure that those already enrolled in both studies continue…
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ReCode Therapeutics has raised $80 million in Series A financing to support the preclinical development of its lead targeted RNA therapy for cystic fibrosis (CF). The funding also will be used to support the development of RNA therapies for primary ciliary dyskinesia (PCD), a hereditary condition that impairs the function…
Translate Bio‘s therapy MRT5005 has been given a rare pediatric disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of cystic fibrosis in children, while its ongoing clinical trial investigating the therapy in adult patients faces delays due to the COVID-19…
Eloxx Pharmaceuticals has temporarily paused enrollment in Phase 2 clinical trials of ELX-02 in cystic fibrosis (CF) patients in response to the COVID-19 pandemic. According to Eloxx,…
Problems with the CFTR protein in platelets, blood cells essential to clotting, activated these cells, both driving lung inflammation and injury, and hindering the lungs’ ability to clear bacteria in a mouse models of cystic fibrosis (CF), a study found. Blocking a calcium channel known as TRPC6 returned platelets…
Growing cells in artificial tracheas or lungs is a better approach to model native organs than cells cultured in spheroids or in a plastic filter, according to a study, suggesting that these platforms may be more suitable to study cell-based therapies for lung diseases such as cystic fibrosis (CF).
Pulmonary artery enlargement is linked to pulmonary hypertension (PH) and lower survival among patients with severe cystic fibrosis (CF), according to a recent review of patient records. The study reporting the findings, titled “Pulmonary artery enlargement is associated with pulmonary…
The non-profit organization Claire’s Place Foundation has launched a donation-based COVID-19 Emergency Fund to support the cystic fibrosis (CF) community by raising funds to provide patients and their families with groceries, supplies, and medications during the outbreak. The fund will be available for people with CF and…
Prolonged use of the antibiotic azithromycin resulted in a 37% slower rate of lung function decline in cystic fibrosis (CF) patients with persistent Pseudomonas aeruginosa infection over a three-year period, a study found. The study, “Pulmonary Outcomes Associated with Long-Term Azithromycin Therapy in Cystic…
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…
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