lysosomal storage disorders

There are more than 50 lysosomal stor­age disorders. Most of them are inherited as autosomal recessive genetic defects. The late onset and slow progression of disease are the specific features of lyso - somal storage disorders. The disorders manifest with muscle weakness, develop­mental delays. Later clinical features are: organomegaly (especially liver and spleen, heart), coarse facial features, joint or skeletal deformities. Usually, no metabolic decompensation occurs.