Mucopolysaccharidoses: The Biochemical Approach

Abstract
Biochemical methods are differentiating these inherited anomalies in terms of the particular factor missing or defective in the cells of the affected individual. This approach has already shown that cells from individuals with almost any one of the syndromes (or from normals) will correct the abnormal mucopolysaccharide metabolism of cells from individuals with one of the other conditions. The implications for therapy are far-reaching indeed.

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