Although curative opportunities are scarce, emerging evidences of new therapeutic strategies, including gene transfer in X-linked myotubular myopathy, enzyme replacement in MTMR14-related congenital myopathies, anti-atrophy approaches, in addition to upregulation of the fetal isoform of skeletal muscle a-actin in patients with recessive mutations in ACTA1 [66, 117] open new chances and challenges. The gene discussed is MTMR14; the disease is congenital myopathy.