[Court-ordered access to treatment of rare genetic diseases: Fabry Disease in the state of Rio Grande do Sul, Brazil].

Court-ordered access to high-cost drugs for rare genetic diseases, such as Fabry Disease (alpha-galactosidase-A deficiency), is a growing phenomenon as yet lacking systematic study. An observational, cross-sectional and retrospective study was conducted to characterize the lawsuits related to access to treatment for Fabry Disease by Enzyme Replacement Therapy in the State of Rio Grande do Sul prior to 2007. The study identified 13 lawsuits and 17 plaintiffs, 11 requesting alfa and 6 betagalsidase.

[High cost drugs for rare diseases in Brazil: the case of lysosomal storage disorders].

This paper approaches in a critical way aspects of Brazilian public policies for drugs, emphasizing those classified as high cost and for rare diseases. The lysosomal storage diseases was taken as an example because of their rarity and the international trend for the development of new drugs for their treatment, all at high costs. Three lysosomal storage diseases were approached: Gaucher disease, Fabry disease and mucopolysaccharidosis type I.

Infections after liver transplantation in adults: data from a university hospital in southern Brazil (1996-2000).

Background: Infections after liver transplantations are the most important cause of morbi-mortality. In this study, we assessed the main characteristics of these infections in a southern Brazilian university hospital.

Methods: We conducted a retrospective cohort with 55 patients who underwent orthotopic liver transplantation between 1996 and 2000 in the "Hospital de Clínicas de Porto Alegre", Porto Alegre, RS, Brazil, to characterize the infections that occurred in the group.