EXPERT REACTION: Aussie trial offers new hope to cystic fibrosis patients
An Australian-led trial has found that adding a new drug, known as VX-445, to two existing treatments significantly improved the lung function of patients with cystic fibrosis. This new drug helped patients who had both one and two copies of the most comment gene defect, which means they have the potential to treat the underlying cause of the disease in 90 per cent of patients. An accompanying editorial describes the trial and a second similar trial as major breakthroughs in cystic fibrosis treatment.
Journal/conference: NEJM
Link to research (DOI): 10.1056/NEJMoa1807120
Organisation/s: The University of Queensland, Monash University, Alfred Hospital, Mater Hospital, National Jewish Health, USA
Funder: Supported by Vertex Pharmaceuticals, which received funding from the Cystic Fibrosis Foundation for the development of VX-445.
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Expert Reaction
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