Gaucher Disease in Iraqi Children.

      Аннотация: Gaucher disease (GD), the most common inherited lysosomal storage disorder, is a multiorgan disease due to an autosomal recessive defect of the gene encoding glucocerebrosidase enzyme. Our aim in this study is to spot light on the clinical and laboratory characteristics of children with Gaucher disease and to evaluate the outcome of enzyme replacement therapy in Iraq, As most of cases in the developing countries still under diagnosed.

Ключевые слова: Gaucher disease, Cerezyme, Therapy, Enzyme replacement