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Hypoglycemic episodes can result in convulsions and brain damage.[15] Untreated episodes of hypoglycemia may progress to coma and death.[8] Metabolic abnormalities can cause hepatic dysfunction that also may progress to hepatic encephalopathy culminating in death.[15] Cardiac abnormalities include cardiomyopathy, which can be fatal, especially during the neonatal period or early childhood

Successful outcomes depend on proper pre-treatment screening, active management of side effects, realistic expectations, and a clear plan for long-term maintenance, whichever path or combination a patient and their physician choose

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