Transgene expression efficacy driven by the Des promoter was successfully demonstrated in 2014 in preclinical studies performed in murine and canine models of X-linked myotubular myopathy (XLMTM) [163] and has recently shown promising results in a clinical trial with meaningful improvements in neuromuscular and respiratory functions (NCT03199469) [164]. This evidence concerns the gene DES and X-linked myotubular myopathy.