Identification of two subtypes of infantile acid maltase deficiency.

Slonim, A E; Bulone, L; Ritz, S; Goldberg, T; Chen, A; Martiniuk, F · J Pediatr · 2000

case_series · Level IV

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Abstract

Infantile patients with acid maltase deficiency have severe hypertrophic cardiomyopathy, left ventricular outflow obstruction, and generalized muscle weakness and die before 1 year of age. We identified 12 infants with acid maltase deficiency who had a similar clinical presentation but less severe cardiomyopathy and absence of left ventricular outflow obstruction, and 9 of 12 had longer survival with assisted ventilation and supplemental intubation.

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