There are times when a single treatment has a significant impact on the overall management of the disease. This is especially true for rare and incurable diseases.

In the field of metabolic diseases, a paradigm shift occurred when researchers found that certain rare patients lacked a single enzyme, which led to the development of therapies to introduce a substitute into the body to fulfill the enzyme’s role.

As a result, patients suffering from these conditions, which are referred to as ‘LSDs (Lysosomal Storage Diseases),’ include Hunter syndrome, Gaucher disease, Fabry disease, and Pompe disease, are currently being treated with enzyme replacement therapy (ERT).

Amon