PMID: 11898513Mar 20, 2002Paper

Pharmacologic and genetic therapy for childhood muscular dystrophies

Current Neurology and Neuroscience Reports
D M Escolar, C G Scacheri

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.

References

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Citations

Jul 30, 2010·Science Translational Medicine·Kumaran ChandrasekharanPaul T Martin
Sep 10, 2003·BMC Neurology·Craig Campbell, Pierre Jacob
Mar 13, 2014·Journal of Muscle Research and Cell Motility·Marcella CantonFabio Di Lisa
Nov 6, 2012·Annual Review of Pathology·Mohammadsharif TabebordbarAmy J Wagers
Feb 12, 2005·Archives of Physical Medicine and Rehabilitation·Gillian A HawkerW Douglas Biggar
Mar 9, 2010·Journal of Child Neurology·Dennis J MatthewsUNKNOWN MD STARnet
Dec 4, 2004·Current Opinion in Rheumatology·Niranjanan NirmalananthanMichael G Hanna
Oct 30, 2012·Journal of Muscle Research and Cell Motility·Jong-Hee KimJohn M Lawler

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