Juan Francisco Cabello, Rodrigo Salinas
Journal: Journal of inherited metabolic disease 2026;49(2):e70152
PMID: 41621834
People living with inborn errors of metabolism (inherited metabolic diseases, IMDs) rely on lifesaving orphan drugs-therapies often developed and available primarily in high-income countries. Yet in many low- and middle-income regions, especially Latin America, patients face stark inequities in access. While an estimated 300 million people worldwide live with a rare disease, most Latin American countries until recently had little to no rare disease policy infrastructure [1]. This perspective highlights the disparity in IMD drug access between wealthy nations and Latin America (with Chile as a case in point), examines the ethical imperatives for equitable and timely access, and explores barriers and potential policy solutions to ensure no patient is left behind.
© 2026 SSIEM.
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