Nebulized and intravenous enzyme replacement therapy in mice with mucopolysaccharidosis type II.

A recent study explored a new method of administering enzyme replacement therapy for mucopolysaccharidosis type II, a hereditary lysosomal storage disease. The study focused on the use of a nebulizer in combination with intravenous treatment to improve delivery of the enzyme idursulfase to the lungs. While the combination treatment increased enzyme activity in the liver, it did not lead to sustained increase in enzyme activity in the lungs. However, it did show persistent modifications in the glycosaminoglycan degradation pathway, suggesting potential additional benefits to intravenous administration alone. This study has not yet undergone peer review. [Extracted from the article]