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The Natural History of Type 1 Infantile GM1 Gangliosidosis: A Literature-Based Meta-Analysis

INTRODUCTION: Type 1 GM1 gangliosidosis is an ultra-rare, rapidly fatal lysosomal storage disorder, with life expectancy of less than 3 years of age. To date, only one prospective natural history study of limited size has been reported. Thus, there is a need for additional research to provide a bett...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab
Main Authors: Lang, Frederick M., Korner, Paul, Harnett, Mark, Karunakara, Ajith, Tifft, Cynthia J.
Formato: Artigo
Idioma:Inglês
Publicado em: 2019
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7093236/
https://ncbi.nlm.nih.gov/pubmed/31937438
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2019.12.012
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