Journal of Pediatrics 2004-Apr
Cerebral edema associated with betaine treatment in classical homocystinuria.
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A child with cystathionine beta-synthase deficiency developed cerebral edema 4 to 6 weeks after starting betaine therapy. There was no evidence of intracranial thrombosis, but there was widespread edema of the white matter. He recovered fully after emergency decompressive craniotomy and withdrawal of betaine.