Lysosomal storage diseases

Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized by lysosomal dysfunction, most of which are inherited as autosomal recessive traits. These disorders are individually rare but collectively affect 1 in 5,000 live births. LSDs typically present in infancy and ch...

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Bibliographic Details
Main Authors: Platt, F, D'Azzo, A, Davidson, B, Neufeld, E, Tifft, C
Format: Journal article
Language:English
Published: Springer Nature 2018