Therapeutic Advances and Novel Drug Treatment Opportunities in the Neuromuscular Disorders Area

Corrado Angelini

Abstract

Several molecular advances have transformed the treatment landscape in the field of neuromuscular disorders, bringing more genetic testing for screening, better biomarkers, new innovative therapies that target specific disease pathways and mechanisms, and a multidisciplinary approach to care. The field includes both genetic and acquired diseases, so the landscape is at various opportunity stages. These advancements have led to more precise diagnoses and personalized medicine in the management of neuromuscular disorders, which can be life-altering for patients. This Editorial examines how the emergence of new therapies impacts the survival and motility of children and adults with spinal muscular atrophy and Duchenne or Becker muscular dystrophy, limb girdle muscular dystrophy, late-onset glycogenosis type 2, myotonic dystrophy and facioscapulohumeral dystrophy. Advances over the past two decades have been substantial in myasthenia gravis (MG) the most common acquired neuromuscular transmission disorder. Despite the existence of few refractory cases, the goal of treatment is the complete remission of symptoms, achieved by thymectomy, immunosuppression, intravenous immunoglobulin, or monoclonal antibody. Advances have been substantial both for serum-positive and serum-negative MG patients who are benefitting from an expansion of treatments since more therapies are available. Also, in the field of metabolic disorders, the use of diet supplements and exercise appears important.

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