FKRP and neuromuscular disease caused by qualitative or quantitative defects of alpha-dystroglycan: To the best of our knowledge, this is the first comprehensive metabolomics study of an FKRP-related dystroglycanopathy animal model, providing extensive detail about the metabolic alterations that occur in the dystrophic muscle as a result of disease progression and in response to therapeutic intervention with FKRP gene therapy.