RESUMO

Mucopolysaccharidosis VI is an autosomal recessive lysosomal storage disorder associated with severe disability and premature death. The presence of a mucopolysaccharidosis-like disease in indigenous ethnic groups in Colombia can be inferred from archaeological findings. There are several indigenous patients with mucopolysaccharidosis VI currently receiving enzyme replacement therapy. We discuss the ethical and economic considerations, regarding both direct and indirect costs, of a high-cost orphan disease in a marginalised minority population in a developing country.

Assuntos

Terapia de Reposição de Enzimas , Mucopolissacaridose VI/economia , N-Acetilgalactosamina-4-Sulfatase/administração & dosagem , Doenças Raras/economia , Colômbia , Pessoas com Deficiência , Terapia de Reposição de Enzimas/economia , Etnicidade , Humanos , Grupos Minoritários , Mucopolissacaridose VI/tratamento farmacológico , Mucopolissacaridose VI/enzimologia , Mucopolissacaridose VI/etnologia , Doenças Raras/etnologia