Answer
Alglucosidase alfa (Myozyme) is thought to restore lysosomal acid α-glucosidase activity, stabilising or restoring cardiac and skeletal muscle function, including respiratory muscles; central nervous system uptake is unlikely because of the blood-brain barrier and the enzyme’s size.
Sources for this answer
- Myozyme 50 mg, powder for concentrate for solution for infusionSANOFI · emc · SmPC §5.1 Pharmacodynamic properties · Version 29Document updated
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