2014•Best Practice & Research Clinical Endocrinology & MetabolismRequires access

The attenuated/late onset lysosomal storage disorders: Therapeutic goals and indications for enzyme replacement treatment in Gaucher and Fabry disease

Carla E. M. Hollak, Neal J. Weinreb

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Key concepts: Enzyme replacement therapy, Fabry disease, Lysosomal storage disease, Disease, Lysosomal storage disorders, Medicine, Context (archaeology), Mucopolysaccharidosis

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The attenuated/late onset lysosomal storage disorders: Therapeutic goals and indications for enzyme replacement treatment in Gaucher and Fabry disease — Research Paper | ScholarLens