Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics
Author:
Affiliation:
1. Telethon Institute of Genetics and Medicine, Naples, Italy
2. Department of Pediatrics, Federico II University, Naples, Italy
Publisher
EMBO
Subject
Molecular Medicine
Link
https://onlinelibrary.wiley.com/doi/pdf/10.1002/emmm.200900036
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4. Replacement Therapy for Inherited Enzyme Deficiency — Macrophage-Targeted Glucocerebrosidase for Gaucher's Disease
5. The pharmacological chaperone 1-deoxygalactonojirimycin increases α-galactosidase A levels in Fabry patient cell lines
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