Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
6154569 | La Presse Médicale | 2014 | 11 Pages |
Abstract
Other strategies using small molecules are being explored in order to cross the blood-brain barrier. This includes substrate reduction or depletion therapies, which decrease the amount of substrate, and the use of pharmacological chaperones, which enhance the residual activity of the mutant enzyme. Miglustat is the proposed substrate reduction therapy in Niemann-Pick C disease and clinical trials are actually performed in several LSD using other substrate reduction or chaperone drugs.
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Authors
Pierre Kaminsky, Olivier Lidove,