Current pharmacologic management in selected neuromuscular diseases.

Andrew J Skalsky, Bjorn Oskarsson, Jay J Han, David Richman

Journal: Physical medicine and rehabilitation clinics of North America 2013;23(4):801-20

PMID: 23137738

Full Text on Elsevier Science View on PubMed

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.

Address: Department of Pediatrics, Rady Children's Hospital San Diego, University of California San Diego, San Diego, CA 92123, USA. [email protected]

DOI: 10.1016/j.pmr.2012.09.003