| Abstrakt: |
This patent application presents a pharmaceutical formulation for treating Pompe disease, a lysosomal storage disorder. The formulation contains a recombinant acid alpha-glucosidase enzyme produced in Chinese hamster ovary cells, along with buffers and excipients. The goal is to maintain enzyme activity and concentration during storage and transportation. The application explores various modifications to the formulation, such as adjusting enzyme concentration and pH levels. This research addresses the need for effective and efficient formulations for enzyme replacement therapy in Pompe disease. [Extracted from the article] |
