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A prospective natural history study of Krabbe disease in a patient cohort with onset between 6 months and 3 years of life
BACKGROUND: Krabbe disease is a rare neurodegenerative disorder caused by a deficiency in the lysosomal enzyme galactocerebrosidase. Patients with Krabbe disease present with a variable disease course depending on their age of onset. The purpose of this prospective cohort study was to characterize t...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
BioMed Central
2018
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6083585/ https://www.ncbi.nlm.nih.gov/pubmed/30089515 http://dx.doi.org/10.1186/s13023-018-0872-9 |