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Mo Med. 2004 Mar-Apr;101(2):100-4.

Enzyme replacement therapy for lysosomal storage disorders: successful transition from concept to clinical practice.

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  • 1Edward A. Doisy Department of Biochemistry and Molecular Biology, Saint Louis University School of Medicine, USA.


Lysosomal disorders offer a striking example of how basic scientific research has been translated into effective medical intervention. The successful application of enzyme replacement therapy for several lysosomal disorders in animal models and in human disease offers hope for everexpanding abilities to counter the effects of these disorders.

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