Nebulized and intravenous enzyme replacement therapy in mice with mucopolysaccharidosis type II
Alex J. Shamoun, Gisienne Reis, Malaica Ashley, Anatália Labilloy, Leonardo F. Ferreira
This study evaluates nebulized and intravenous enzyme replacement therapy in mice with mucopolysaccharidosis type II, finding that combination treatment improves liver enzyme activity and disease features but not lung enzyme activity.
PLoS ONE · Jun 26, 2025Peer-reviewedGenetics