title: Current pharmacologic management in selected neuromuscular diseases.
abstract: For generations, the neuromuscular disorder care community has focused on establishing the correct diagnosis and providing supportive care. As the pathophysiology and genetics of these conditions became better understood, novel treatments targeting the disease mechanism were developed. This has led to some significant disease-modifying and supportive treatments for several neuromuscular disorders. The current treatments for amyotrophic lateral sclerosis (ALS), neuromuscular junction disorders, inflammatory myopathies, and myotonia are reviewed. Additionally, investigational treatments for ALS, Duchenne muscular dystrophy, and spinal muscular atrophy are discussed.
authors.affiliation: Department of Pediatrics, Rady Children's Hospital San Diego, University of California San Diego, San Diego, CA 92123, USA. askalsky@rchsd.org
authors.forename: Andrew J
authors.initials: AJ
authors.lastname: Skalsky
authors.forename: Bjorn
authors.initials: B
authors.lastname: Oskarsson
authors.forename: Jay J
authors.initials: JJ
authors.lastname: Han
authors.forename: David
authors.initials: D
authors.lastname: Richman
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000150:complications / Q000188:drug therapy; D018691:Excitatory Amino Acid Antagonists / Q000627:therapeutic use; D006009:Glycogen Storage Disease Type II / Q000188:drug therapy*; D006801:Humans; D009134:Muscular Atrophy, Spinal / Q000188:drug therapy; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy; D009220:Myositis / Q000188:drug therapy; D009222:Myotonia / Q000188:drug therapy; D009468:Neuromuscular Diseases / Q000188:drug therapy*; D020511:Neuromuscular Junction Diseases / Q000188:drug therapy
chemical_list: D018691:Excitatory Amino Acid Antagonists