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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease

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Fabry disease - current treatment and new drug development

Omid Motabar1, Ellen Sidransky, Ehud Goldin

  • 1Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, 35 Convent Drive, MSC 3708, Bethesda, MD 20894-3708, USA.

Current Chemical Genomics
|December 4, 2010
PubMed

Keywords:
Alpha-GalactosidaseChaperone TherapyDrug Development.Fabry DiseaseHigh Throughput ScreeningLysosomal Storage Disorders

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