At large, the rare disease community is starting to see hope from the potential of gene therapy, with hundreds of studies and A study conducted in humans that evaluates the effectiveness, safety, and side effects of investigational products (which could include both medications, treatments, or devices) that have the possibility of treating a disease in that particular patient group. currently enrolling for gene therapy across a number of genetic conditions. In fact, progress has been made in treatment of other lysosomal diseases using gene therapy. Scientists and researchers can lean on this knowledge and apply it to the A protein responsible for speeding the rate of chemical reaction in the cell of the living organism. Enzymes support a wide range of functions in an organism, as they spark chemical reactions. Enzymes work with, or interact with, substrates (the underlying surface or substance). When reacted together, the enzyme and substrate result in a new product or molecule that then separates from the original enzyme and goes on to create new reactions in the cell. replacement therapy process for AGU as the current standard of care.