In March, the U.K. National Institute for Health and Care Excellence (NICE) issued a draft guidance against recommending Orkambi (lumacaftor-ivacaftor marketed by Vertex Pharmaceuticals) for treating cystic fibrosis (CF). The agency’s independent appraisal committee concluded that the cost of Orkambi was considerably higher than the current standard of care, and it could not be…
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ProQR Therapeutics is set to release results from a study on the effect of the investigational drug QR-010 on nasal potential difference – an evaluation of CFTR protein function in patients with cystic fibrosis (CF). The findings will be presented at the North American Cystic Fibrosis Conference (NACFC), Oct. 27…
International Biophysics Corporation recently received CE Mark, Health Canada, and Australian TGA approval for AffloVest, allowing the company to sell and market the vest in Canada, Australia and New Zealand, and in countries using the CE mark. The AffloVest is a portable high frequency chest wall oscillation (HFCWO) vest that promotes airway…
Hill-Rom Holdings recently announced the introduction of the VisiVest System – a connected therapeutic garment for patients requiring airway clearance therapy, including patients with cystic fibrosis (CF). The VisiVest System combines innovative high-frequency chest-wall oscillation (HFCWO) technology with wireless connectivity powered by Qualcomm Life. The HFCWO technology consists…
To date, 2,007 mutations have been identified in the CFTR gene, but their contribution for cystic fibrosis (CF) development has not been defined for the majority of these mutations. Now, researchers at Children’s Hospital Los Angeles and the Genetic Disease Screening Program of the California Department of Public Health have…
Alcresta Therapeutics recently launched the commercialization of the digestive enzyme cartridge Relizorb to help patients unable of breaking down fats, particularly adults with cystic fibrosis (CF) who depend on enteral nutrition. Cystic fibrosis is a life-threatening genetic condition caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene…
Vertex Pharmaceuticals recently revealed new clinical data on real-world long-term outcomes in cystic fibrosis (CF) patients treated with Kalydeco (ivacaftor). The company also presented data from a Phase 3 study evaluating Orkambi (lumacaftor/ivacaftor) for children with CF ages 6-11 years. The results were available June 11-14 at the 39th European Cystic Fibrosis Society (ECFS)…
Raptor Pharmaceutical recently announced new clinical data from a Phase 3 trial evaluating the efficacy of Quinsair (levofloxacin) versus tobramycin in cystic fibrosis (CF) patients with chronic pulmonary infections caused by Pseudomonas aeruginosa. Study results were presented at the 39th European Cystic Fibrosis Conference in Basel, Switzerland. “Patients with cystic fibrosis have few…
TGV-inhalonix reported clinical findings at the European Cystic Fibrosis Conference recently Basel, Switzerland, that show the potential of a new drug to treat mixed bacterial and fungal lung infections contracted by patients with cystic fibrosis (CF). The data presented by George Tetz, scientific and development advisor of TGV-inhalonix, indicated that Mul-1867…
Researchers at the University of Iowa have uncovered a link between the dysfunction of cystic fibrosis protein CFTR and airway smooth muscle (ASM) abnormalities — two common pathologies of the chronic lung disorder cystic fibrosis (CF). The study, “Acute administration of ivacaftor to people with cystic fibrosis and a…
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