Advances in Treatments in Muscular Dystrophies and Motor Neuron Disorders.

Increased understanding of disease pathophysiology and advances in gene therapies and drug technologies are revolutionizing treatment of muscular dystrophies and motor neuron disorders (MNDs). New drugs have been approved for Duchenne muscular dystrophy, spinal muscular atrophy, and amyotrophic lateral sclerosis. For other diseases, new targets have been identified, and new therapies are in clinical trials. The impact of such therapies will be fully understood only in the next decades. Cost burden and accessibility are major challenges in the wide application of new drugs. This article reviews advances in gene therapies, newly approved drugs, and therapeutic promises in muscular dystrophies and MNDs.
Competing Interests: Disclosure Dr B. Roy has served as a consultant/advisor for Alexion Pharmaceuticals. Dr R. Griggs has received grant funding from Muscular Dystrophy Association (MDA), Muscular Dystrophy Association (NIH), Parent Project Muscular Dystrophy (PPMD), PTC therapeutics (PTC), and Sarepta BioPharma. He also has served as a consultant for PTC, Sarepta, and Strongbridge Biopharma, and on a Data and Safety Monitoring Board (DSMB) for Solid Biosciences.
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