Publications by authors named "Claudia Leralta-Gonzalez"
An Pediatr (Engl Ed)
January 2022
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.
An Pediatr (Engl Ed)
November 2020
Article Synopsis
- Inborn errors of metabolism are rare diseases that require specialized treatments like diet therapy and enzyme replacement, with limited patient data available.
- A retrospective study of pediatric patients with these conditions evaluated the effectiveness of nine different orphan drugs over 24 months at a hospital, showing varied effects on patients' symptoms.
- While most patients tolerated the treatments well, concerns remain about the high costs and uncertain cost-effectiveness of these orphan drugs given their mixed effectiveness and significant financial impact on healthcare.
