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Pharmacologic and genetic therapy for childhood muscular dystrophies

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Abstract

The outstanding advances in the molecular characterization of muscle diseases, including muscular dystrophies, inflammatory myopathies, and ion channel disorders, have resulted in the identification of potential targets for pharmacologic and genetic therapy in the best characterized of these diseases. The most common myopathy in children, Duchenne muscular dystrophy (DMD), is the focus of active pharmacologic clinical trials. Genetic transfer therapy research for this and other dystrophies is rapidly moving forward. However, as new approaches for treatment are being actively investigated, the current modality of treatment for all myopathies is still in the realm of physical medicine and rehabilitation. The focus of this review is on the advances in pharmacologic and genetic therapy research in DMD and limb girdle muscular dystrophies.

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Authors and Affiliations

  1. Department of Neurology, Research Center for Genetic Medicine, MDA Clinic, Children’s National Medical Center, George Washington University, 111 Michigan Avenue NW, 20010, Washington, DC, USA

    Diana M. Escolar MD

  2. Research Center for Genetic Medicine, Children’s National Medical Center, 111 Michigan Avenue NW, 20010, Washington, DC, USA

    Cheryl G. Scacheri MS, CGC

Authors
  1. Diana M. Escolar MD
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  2. Cheryl G. Scacheri MS, CGC
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Escolar, D.M., Scacheri, C.G. Pharmacologic and genetic therapy for childhood muscular dystrophies. Curr Neurol Neurosci Rep 1, 168–174 (2001). https://doi.org/10.1007/s11910-001-0013-y

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  • DOI: https://doi.org/10.1007/s11910-001-0013-y

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