Registries for rare diseases are broadly accepted for their usefulness in obtaining epidemiological data and patient recruitment for clinical trials [10] Translational Research in Europe–Assessment and Treatment of Neuromuscular Diseases (TREAT-MND ALLIANCE), a research network for neuromuscular disorders, developed a global database for patients with Duchenne muscular dystrophy (DMD) [11], spinal muscular atrophy, alpha-dystroglycanopathy with mutations in FKRP, and dysferlinopathy [12]. This evidence concerns the gene FKRP and Duchenne muscular dystrophy.