In school, the classes could be put together in an optimal fashion to keep the patient in a safe and caring environment. The society could be helpful in organizing leisure activities or protected work to improve the quality of life. Building programs for wheel-chair adapted houses equipped with elevator could secure the diseased an independent life. When exogenous lysosomal enzymes are injected into the patient, this enzyme can eventually be taken up by enzyme-deficient cells. This uptake is regulated by certain receptors on the cell surface.
Different tissue have different receptors.
It is therefore necessary to change the natural enzyme to enable uptake in the tissue of interest. If properly designed, the missing enzyme could be injected regularly like the diabetic subject injects insulin. H owever, it took 15 years after the publication of the paper Toward Enzyme Therapy for Lysosomal Storage Diseases before the first patients with Gaucher disease were treated successfully. This reflects the time lag between idea and reality. However, compared to other lysosomal storage diseases, the prerequisite for enzyme replacement are especially favorable in Gaucher, since the accumulation of storage material is restricted to the cells which preferably take up the substituted enzyme.