Celtaxsys has completed its Phase 2b trial assessing the safety and effectiveness of acebilustat (formerly CTX-4430) for cystic fibrosis (CF), with the company announcing the last visit of the last enrolled patient. Details of the trial, supported by the Cystic Fibrosis Foundation, will be presented at the upcoming 41st…
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An antibacterial compound found in toothpaste, called triclosan, may be part of a future cystic fibrosis (CF) treatment, a Michigan State University study suggests. The research, “Triclosan is an aminoglycoside adjuvant for the eradication of Pseudomonas aeruginosa biofilms,” was published in the journal Antimicrobial Agents…
A baby born with cystic fibrosis (CF) in Germany today is likely to live to be 40 or 50, while another born in the Ukraine will probably die before the age of 18. That’s the appraisal of Hilde De Keyser, coordinator of Cystic Fibrosis Europe — a Brussels-based federation that…
The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…
Cystic fibrosis (CF) patients showed improved clinical outcomes and less disease progression after long-term treatment with Kalydeco (ivacaftor), according to an observational study. Researchers found that Kalydeco-treated CF patients had a significantly lower risk of death, pulmonary exacerbation, hospitalization, transplant, and prevalence of CF-related complications and pathological microorganisms,…
Tobramycin, an antibiotic often used to treat acute lung infections in cystic fibrosis patients, is safe for once-a-day use in children under age 6, with findings of minimal kidney and no inner ear toxicity in these pediatric patients, a study reports. The research, “Safety of Extended Interval Tobramycin…
Patients with cystic fibrosis (CF) are at higher-than-average risk of developing gastrointestinal cancers, especially those who underwent a lung transplant, a new study shows. Transplant patients with CF were found in this retrospective study to have a five-times greater risk of gastrointestinal cancer than those who have not had a…
A team of professional cyclists are riding six custom-designed and hand-painted bicycles — each inspired by a child whose medical needs were supported by the UnitedHealthcare Children’s Foundation (UHCCF) — at the 2018 Amgen Tour of California now underway. The Rally Cycling team’s goal is to help raise money…
An advisory committee composed by experts in the field of cystic fibrosis will help to guide the design of the global Phase 3 program testing PTI-428, the company developing the potential treatment, Proteostasis Therapeutics, announced. Its formation is a key step in ensuring that the program follows regulatory requirements, while retaining…
The approved CFTR modulators developed by Vertex Pharmaceuticals are too expensive for the clinical benefit they can provide to cystic fibrosis (CF) patients, according to the Institute for Clinical and Economic Review (ICER). ICER evaluated the effectiveness and costs of Symdeko (tezacaftor/ivacaftor), Orkambi (lumacaftor/ivacaftor), and…
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