Has gene therapy been successful in diseases similar to AGU?

At large, the rare disease community is starting to see hope from the potential of gene therapy, with hundreds of studies and clinical trials 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 enzyme replacement therapy process for AGU as the current standard of care.