Abstract
Objective: To identify and disseminate the challenges faced by patients with Laron syndrome in accessing Mecasermin treatment in Ecuador. Theoretical Framework: Laron syndrome is a rare genetic disease characterised by metabolic disorders such as hypoglycemia and severe growth retardation. Treatment with Mecasermin improves growth and thus quality of life for those affected. Ecuador has the largest Laron syndrome population in the world, extensively studied contributing to understanding the peculiarities and needs of these patients. Method: Official documents, letters, news articles from national and international press, and legal and medical databases were reviewed. Legal and medical advice was sought for this study. Results and Discussion: Over approximately ten years, a group of parents pursued national and international legal action to provide their children with the only scientifically validated treatment for this syndrome. However, authorities denied these requests without justification. It was only after the Inter-American Commission on Human Rights report the admissibility of this case that Ecuador began administering medication to children with Laron syndrome. Research Implications: Increase public awareness of this rare disease and barriers faced by those affected, fostering empathy, and understanding of patients' needs. Originality/Value: Identification of specific barriers to accessing treatment, while the quality of life of patients and their families is affected.
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