This has led to the suggestion that the administration of pharmacologically active small molecules, capable of upregulating LARGE, or LARGE2 which however is essentially not expressed in muscle, or both, could be an interesting therapeutic strategy for a broad range of dystroglycanopathy patients [30], [31], [34]. This evidence concerns the gene LARGE1 and neuromuscular disease caused by qualitative or quantitative defects of alpha-dystroglycan.