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Therapeutic Approaches for the Sarcomeric Protein Diseases

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The Sarcomere and Skeletal Muscle Disease

Part of the book series: Advances in Experimental Medicine and Biology ((AEMB,volume 642))

Abstract

No curative treatment currently exists for patients with skeletal myopathies caused by defects in sarcomeric proteins though, symptomatic treatments including orthoses, night-time ventilation, or mechanical ventilation can provide major benefits. The molecular genetic discovery era has enabled many families to know which gene and precisely which gene defect their family, or in some cases only their affected child has. This knowledge has enormously increased the accuracy of genetic counselling and in some cases can enable prognosis, which helps families to make better-informed life decisions. However, symptomatic treatments and molecular genetics do not help the patient’s skeletal muscle problems. The patients with skeletal muscle sarcomeric protein diseases, (from severely affected patients with shortened lifespan, through to the more mildly affected patients), would all benefit from more effective or curative treatments, as would their parents and families. This chapter outlines the experimental therapeutic strategies that have been investigated for other muscle diseases (predominantly the muscular dystrophies, towards which the majority of research emphasis has been focussed) and those that are beginning to be investigated for sarcomeric diseases. It analyses which of these approaches might be applicable to the different skeletal muscle sarcomeric protein diseases.

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  1. Centre for Medical Research, University of Western Australia, Western Australian Institute for Medical Research, B Block, QEII Medical Centre, Nedlands, Western Australia, 6009, Australia

    Kristen J. Nowak

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  1. Centre for Medical Research, University of Western Australia, Western Australian Institute for Medical Research, Nedlands, Western Australia, Australia

    Nigel G. Laing PhD

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Nowak, K.J. (2008). Therapeutic Approaches for the Sarcomeric Protein Diseases. In: Laing, N.G. (eds) The Sarcomere and Skeletal Muscle Disease. Advances in Experimental Medicine and Biology, vol 642. Springer, New York, NY. https://doi.org/10.1007/978-0-387-84847-1_15

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