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In Vitro and In Vivo Amenability to Migalastat in Fabry Disease

Malte Lenders, Franciska Stappers, Eva Brand

2020Molecular Therapy — Methods & Clinical Development42 citationsDOIOpen Access PDF

Abstract

amenability, the treating clinician has the responsibility to monitor clinical and laboratory features to verify clinical response.

Topics & Concepts

Fabry diseaseIn vivoIn vitroMedicineMutationComputational biologyDiseaseBioinformaticsBiologyGeneticsInternal medicineGeneLysosomal Storage Disorders ResearchCarbohydrate Chemistry and SynthesisTrypanosoma species research and implications