The juvenile gangliosidoses: A timeline of clinical change

Background: The gangliosidoses are rare inherited diseases that result in pathologic accumulation of gangliosides in the central nervous system and other tissues, leading to severe and progressive neurological impairment and early death in the childhood forms. No treatments are currently approved fo...

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Bibliographic Details
Main Authors: Kelly E. King, Sarah Kim, Chester B. Whitley, Jeanine R. Jarnes-Utz
Format: Article
Language:English
Published: Elsevier 2020-12-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426920301221