We specialize in advancing and supporting the development of a variety of therapies for genetic disorders of children, including gene therapies and protein replacement strategies. We have traditionally worked on skeletal muscle diseases but have now expanded to work in essentially all other tissue types. Our work also involves supporting therapeutic development and safety programs spanning from the early preclinical to the human clinical trial stage. Our translational science approach is applicable to a broad range of therapeutic approaches, as the techniques and expertise necessary to judge efficacy and safety is not restricted to a given disease state or treatment type.