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Authors
Advisor(s)
Abstract(s)
Invited speaker at Med-Lysosomal2019. Following the success of three earlier meetings on rare diseases wherein LSDs were important components, the meeting in London focused on the diagnosis and treatment as well as on new therapeutic modalities already in advanced stages of development of four main Lysosomal Diseases: Gaucher, Fabry, Pompe and MPSs.
Description
Invited speaker
Keywords
Human Genetics Gene Editing CRISPR/Cas Cell Models Doenças Genéticas