Cardiac involvement in Sandhoff's disease. Inborn error of glycosphingolipid metabolism

Leonard C. Blieden, Robert J. Desnick, John B. Carter, William Krivit, James H. Moller, Harvey L. Sharp

Research output: Contribution to journalArticlepeer-review

40 Scopus citations

Abstract

Two siblings with Sandhoff's disease manifested clinical and laboratory evidence of significant cardiomyopathy. Postmortem examination of the hearts of both patients revealed anatomic, biochemical and ultrastructural abnormalities resulting from deficient activities of the enzymes hexosaminidase A and B. A study of the biochemical and ultrastructural changes in the hearts revealed the accumulation of the glycosphingolipid globoside in all cardiac tissues. The necropsy findings indicate that Sandhoff's disease should be considered in the differential diagnosis in infants with cardiomyopathy or mitral insufficiency.

Original languageEnglish
Pages (from-to)83-88
Number of pages6
JournalAmerican Journal of Cardiology
Volume34
Issue number1
DOIs
StatePublished - Jul 1974
Externally publishedYes

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