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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...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7674119/ https://www.ncbi.nlm.nih.gov/pubmed/33240792 http://dx.doi.org/10.1016/j.ymgmr.2020.100676 |