Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial -

Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
Information type: Randomised controlled trials
Source: The Lancet Respiratory Medicine
Specialities: Paediatric and neonatal medicine | Respiratory disorders
Summary
Monthly application of the pGM169/GL67A gene therapy nebulised formulation was associated with a significant, but modest benefit in FEV1 compared with placebo at 1 year (n=116, p=0.046), and no difference in AEs, indicating a stabilisation of lung function in the treatment group.
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