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Orphanet Journal of Rare Diseases, ISSN 1750-1172, 2012, Volume 7, Issue 1, pp. 32 - 32
Journal Article
2013, ISBN 0444595651, Volume 113
Lysosomal storage diseases (LSD) are inborn errors of metabolism secondary to lysosomal enzyme defects and are characterized by a progressive accumulation of... 
Enzyme Replacement Therapy | Blood-Brain Barrier - enzymology | Lysosomal Storage Diseases - drug therapy | Child | Humans
Book Chapter
Journal Article
Journal Article
Orphanet Journal of Rare Diseases, ISSN 1750-1172, 09/2014, Volume 9, Issue 1, p. 130
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 09/2015, Volume 373, Issue 11, pp. 1010 - 1020
Journal Article
Journal Article
Orphanet Journal of Rare Diseases, ISSN 1750-1172, 2012, Volume 7, Issue 1, pp. 36 - 36
Journal Article