Publications by authors named "Alicia Caso-Gonzalez"
Article Synopsis
- Inborn errors of metabolism are diverse orphan diseases treated mainly through dietary changes and orphan drugs, but research is limited due to the small patient population.
- A study analyzed the effectiveness of nine different orphan drugs on 15 pediatric patients, revealing mixed results in symptom improvement while showcasing reasonable treatment tolerance.
- The high cost of these drugs raises questions about their cost-effectiveness, especially given the varying effectiveness and limited clinical evidence available.
Introduction: Inborn errors of metabolism are a highly heterogeneous group of orphan diseases. Diet therapy and enzyme and coenzyme replacement are the most frequently used treatment. There are few patients and published studies about inborn errors of metabolism.
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