Method and composition for treating Hunter syndrome

摘要

To provide improved methods and compositions for treating Hunter syndrome.SOLUTION: A pharmaceutical composition for treating Hunter syndrome comprises a therapeutically effective dose of an ICV formulation comprising idursulfase-beta (IDS-β) protein at a concentration ranging from 0.1 mg/ml to 60 mg/ml, sodium chloride at a concentration of 150 mM, polysorbate 20 at a concentration of 0.05 mg/ml, and a pH of 6. The pharmaceutical composition is administered intracerebroventricularly (ICV administration) to a subject in need of treatment.SELECTED DRAWING: Figure 1