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Infantile Gangliosidoses: Mapping a Timeline of Clinical Changes

BACKGROUND: Infantile gangliosidoses include GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs disease, Sandhoff disease). To date, natural history studies in infantile GM2 (iGM2) have been retrospective and conducted through surveys. Compared to iGM2, there is even less natural history informati...

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Détails bibliographiques
Publié dans:Mol Genet Metab
Auteurs principaux: Jarnes Utz, Jeanine R., Kim, Sarah, King, Kelly, Ziegler, Richard, Schema, Lynn, Redtree, Evelyn S., Whitley, Chester B.
Format: Artigo
Langue:Inglês
Publié: 2017
Sujets:
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5727905/
https://ncbi.nlm.nih.gov/pubmed/28476546
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2017.04.011
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