Targeted Therapies for Metabolic Myopathies Related to Glycogen Storage and Lipid Metabolism: a Systematic Review and Steps Towards a ‘Treatabolome’

Background: Metabolic myopathies are a heterogenous group of muscle diseases typically characterized by exercise intolerance, myalgia and progressive muscle weakness. Effective treatments for some of these diseases are available, but while our understanding of the pathogenesis of metabolic myopathie...

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Veröffentlicht in:	Journal of Neuromuscular Diseases 2021-01, Vol.8 (3), p.401-417
Hauptverfasser:	Manta, A., Spendiff, S., Lochmüller, H., Thompson, R.
Format:	Artikel
Sprache:	eng
Schlagworte:	Acyl-CoA dehydrogenase Carnitine Computer applications Disease management Drug therapy Genetic diversity Glycogen Glycogen - metabolism Glycogen Storage Disease Type II - drug therapy Humans Intolerance Lipid Metabolism Lipids Literature reviews Metabolism Metabolism, Inborn Errors - drug therapy Multiple Acyl Coenzyme A Dehydrogenase Deficiency - drug therapy Muscle Weakness Mutation Myalgia Oxidation Riboflavin Supplements Systematic Review Systemic primary carnitine deficiency
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