Resumo
This proposal has two components: the research and educational. The research component focus on Gaucher disease, Gaucher disease is an autosomal recessive disease with a prevalence of 1:60,000 to 1:200,000 births. So far there is no cure, but there is treatment. Current treatments consist of replacement therapy with placenta-derived glucocerebrosidase (pdGBA) or recombinant glucocerebrosi…