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Pharmacology. 2010;85(1):1-17. doi: 10.1159/000259044. Epub 2009 Nov 18.

Amyloidosis and neurodegenerative diseases: current treatments and new pharmacological options.

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  • 1Académie Nationale de Médecine Paris, Paris, France.


Most neurodegenerative diseases share several clinical, genetic and pathophysiological features, and an irreversible evolution as well. They are characterized by an endogenous production of abnormal proteins called amyloid proteins (AP), which are not hydrosoluble, form depots, and are only partly cleared by autophagy and the ubiquitin-protease system. Despite their different structures, they are probably generated by a common pathological pathway, a misfolding process. This hypothesis suggests a common pharmacological approach, which can consist of either the blockade of the misfolding process, the elimination of AP or both. The currently validated treatments are mostly palliative ones, trying to supplant the function of destroyed neurons. New trends involve the regulation of the cerebral cholesterol metabolism and the preservation of neuron mitochondrial functions. Special attention is given to already marketed drugs used for other indications, which are also able to act on neurodegeneration.

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