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NTBC therapy for tyrosinemia type 1: how much is enough
European Journal of Pediatrics, 11/04/09

El–Karaksy H et al. – Doses of NTBC, lower than recommended, may be helpful in treatment of tyrosinemia, on condition that succinylacetone production is suppressed, and AFP is maintained normal or showing a progressive decrease. This cost–effective dose may allow treatment of affected children from economically underprivileged countries, but longer follow up periods are needed.

Significant increase of succinylacetone within the first 12 h of life in hereditary tyrosinemia type 1
European Journal of Pediatrics, 10/13/09 Relevance Score: 80%