On the path to evidence-based therapy in neuromuscular disorders

Neuromuscular disorders encompass a diverse group of acquired and genetic diseases characterized by loss of motor functionality. Although cure is the goal, many therapeutic strategies have been envisioned and are being studied in randomized clinical trials and entered clinical practice. As in all scientific endeavors, the successful clinical translation depends on the quality and translatability of preclinical findings and on the predictive value and feasibility of the clinical models. This chapter focuses on five exemplary diseases: childhood spinal muscular atrophy (SMA), Charcot-Marie-Tooth (CMT) disorders, chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), acquired autoimmune myasthenia gravis (MG), and Duchenne muscular dystrophy (DMD), to illustrate the progress made on the path to evidenced-based therapy.