Gaucher disease: Enzyme therapy in the a
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Prows, Cynthia A.; Sanchez, Nelson; Daugherty, Cynthia; Grabowski, Gregory A.
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Article
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1997
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John Wiley and Sons
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English
β 23 KB
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The responses to regular intravenous enzyme infusions were compared in two sibs with Gaucher disease type 2, the acute neuronopathic variant. Enzyme administration was begun at 7 months in patient 1 who had severe progressive visceral and neuronopathic disease. No significant effect of enzyme infusi