PHARMACEUTICAL COMPOSITION FOR ENZYME REPLACEMENT THERAPY

摘要

Disclosed is a pharmaceutical composition for the treatment of Fabry disease, which has few allergic adverse side effects, has high stability in the blood (plasma), can be taken up readily by a cell in a dysfunctional organ, and utilizes a protein having an a-galactosidase activity. The pharmaceutical composition comprises, for example, a protein which acquires an a-N-galactosidase activity through the alternation of the structure of the active site in wild-type human a-acetylgalactosaminidase.