Lysosomal Hydrolases
Inborn Errors of Metabolism
Glucose Transporters
Pleiotropy
Neural Regulation
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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
Published on: December 20, 2017
Arielle Crespel1, Edoardo Ferlazzo2, Silvana Franceschetti3
1Epilepsy Unit, Hôpital Gui-de-Chauliac, Research Unit 'Movement Disorders' (URMA), Department of Neurobiology, Institute of Functional Genomics, Montpellier, France.
Unverricht-Lundborg disease (ULD) is a progressive myoclonus epilepsy caused by mutations in the cystatin B (CSTB) gene. A mouse model revealed CSTB’s role in neuronal function, offering new therapeutic targets.
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